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ecd:PeoMember 2022-01-01 2022-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:PeoMember 2021-01-01 2021-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:PeoMember 2020-01-01 2020-12-31 0001515673 rare:LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember ecd:PeoMember 2023-01-01 2023-12-31 0001515673 rare:LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember ecd:PeoMember 2022-01-01 2022-12-31 0001515673 rare:LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember ecd:PeoMember 2021-01-01 2021-12-31 0001515673 rare:LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember ecd:PeoMember 2020-01-01 2020-12-31 0001515673 rare:LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember ecd:NonPeoNeoMember 2023-01-01 2023-12-31 0001515673 rare:LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember ecd:NonPeoNeoMember 2022-01-01 2022-12-31 0001515673 rare:LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember ecd:NonPeoNeoMember 2021-01-01 2021-12-31 0001515673 rare:LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember ecd:NonPeoNeoMember 2020-01-01 2020-12-31 0001515673 rare:PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember ecd:NonPeoNeoMember 2023-01-01 2023-12-31 0001515673 rare:PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember ecd:NonPeoNeoMember 2022-01-01 2022-12-31 0001515673 rare:PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember ecd:NonPeoNeoMember 2021-01-01 2021-12-31 0001515673 rare:PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember ecd:NonPeoNeoMember 2020-01-01 2020-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember ecd:NonPeoNeoMember 2023-01-01 2023-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember ecd:NonPeoNeoMember 2022-01-01 2022-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember ecd:NonPeoNeoMember 2021-01-01 2021-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember ecd:NonPeoNeoMember 2020-01-01 2020-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:NonPeoNeoMember 2023-01-01 2023-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:NonPeoNeoMember 2022-01-01 2022-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:NonPeoNeoMember 2021-01-01 2021-12-31 0001515673 rare:PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember ecd:NonPeoNeoMember 2020-01-01 2020-12-31 0001515673 rare:LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember ecd:NonPeoNeoMember 2022-01-01 2022-12-31 0001515673 1 2023-01-01 2023-12-31 0001515673 2 2023-01-01 2023-12-31 0001515673 3 2023-01-01 2023-12-31 0001515673 4 2023-01-01 2023-12-31 iso4217:USD shares iso4217:USD shares pure false 0001515673 DEF 14A ULTRAGENYX PHARMACEUTICAL INC. <h2 id="xdx_80C_eecd--PvpTableTextBlock_dU_z2TdPohEldoh" style="font: 20pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify; color: #7CBC3E"><b><span id="lrarea097"></span>Pay versus Performance</b></h2> <p style="font: 15pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify">As required by Section 953(a) of the Dodd-Frank Wall Street Reform and Consumer Protection Act, and Item 402(v) of Regulation S-K, we are providing the following information about the relationship between Compensation Actually Paid (CAP) and certain financial performance of the Company. For further information concerning the Company’s pay for performance philosophy and how the Company’s aligns executive compensation with the Company’s performance, refer to “Executive Compensation – Compensation Discussion and Analysis.”</p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <table cellpadding="0" cellspacing="0" id="xdx_307_zXJDzuKhXmRk" style="font: 9pt Arial, Helvetica, Sans-Serif; border-collapse: collapse; width: 100%" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="vertical-align: bottom"> <td style="white-space: nowrap; text-align: left; padding-left: 3pt"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" rowspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Average<br/> Summary<br/> Compensation<br/> Table Total for<br/> Non-PEO NEOs⁽³⁾</b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="6" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: center"><b>Value of Initial Fixed $100<br/>Investment Based On:</b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td> <td colspan="2" style="white-space: nowrap"><b> </b></td><td style="white-space: nowrap"><b> </b></td></tr> <tr style="vertical-align: bottom"> <td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: left; padding-left: 3pt"><b>Year</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Summary<br/> Compensation Table<br/> Total for PEO⁽¹⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Compensation<br/> Actually Paid to <br/> PEO⁽²⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Average<br/> Compensation<br/> Actually Paid to<br/> Non-PEO NEOs⁽⁴⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Total<br/> Shareholder<br/> Return⁽⁵⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Peer Group<br/> Total<br/> Shareholder <br/> Return⁽⁶⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Net Income<br/> (Loss)<br/> (in thousands)⁽⁷⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td> <td colspan="2" style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b>Revenue<br/> (in thousands)⁽⁸⁾</b></td><td style="white-space: nowrap; border-bottom: rgb(124,188,62) 2px solid; text-align: right"><b> </b></td></tr> <tr style="vertical-align: bottom; background-color: White"> <td style="width: 28%; font-weight: bold; text-align: left; padding-left: 3pt">2023</td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">      $</td><td id="xdx_987_eecd--PeoTotalCompAmt_c20230101__20231231_fKDEp_zdCommiwdFXd" style="width: 5%; text-align: right">13,077,993</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">  $</td><td id="xdx_985_eecd--PeoActuallyPaidCompAmt_c20230101__20231231_fKDIp_zeXlArVekQ36" style="width: 5%; text-align: right">11,293,489</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">   $</td><td id="xdx_981_eecd--NonPeoNeoAvgTotalCompAmt_c20230101__20231231_fKDMp_zWNzHkNkOiIf" style="width: 5%; text-align: right">4,524,001</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">    $</td><td id="xdx_983_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20230101__20231231_fKDQp_zmI8ksRsrdb1" style="width: 5%; text-align: right">4,829,669</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">    $</td><td id="xdx_98A_eecd--TotalShareholderRtnAmt_pp2d_c20230101__20231231_fKDUp_zQkodFmzV52a" style="width: 5%; text-align: right">112</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">      $</td><td id="xdx_980_eecd--PeerGroupTotalShareholderRtnAmt_pp2d_c20230101__20231231_fKDYp_zZSNCBEsL3lg" style="width: 5%; text-align: right">115</td><td style="width: 1%; text-align: left"> </td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">       $</td><td style="width: 5%; text-align: right"><span id="xdx_905_eus-gaap--NetIncomeLoss_pn3n3_uUSD_c20230101__20231231_fKDcp_ztvR31cAfMe9">(606,639</span>)</td><td style="width: 1%; text-align: left"></td><td style="width: 2%"> </td> <td style="width: 1%; text-align: right">       $</td><td style="width: 5%; text-align: right"><span id="xdx_90C_eecd--CoSelectedMeasureAmt_pn3n3_uUSD_c20230101__20231231_fKDgp_zVMztGY8FsTd">434,249</span></td><td style="width: 1%; text-align: left"> </td></tr> <tr style="vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font-weight: bold; text-align: left; padding-left: 3pt">2022</td><td> </td> <td style="text-align: right">$</td><td id="xdx_980_eecd--PeoTotalCompAmt_c20220101__20221231_fKDEp_zvlZOy1ua6S3" style="text-align: right">12,140,330</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_98B_eecd--PeoActuallyPaidCompAmt_c20220101__20221231_fKDIp_zMyCmlwl4lAd" style="text-align: right">3,311,432</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_98D_eecd--NonPeoNeoAvgTotalCompAmt_c20220101__20221231_fKDMp_zjPEcXzZ1Ao2" style="text-align: right">3,911,773</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_987_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20220101__20221231_fKDQp_zSqij2WZNor9" style="text-align: right">(175,590)</td><td style="text-align: left"></td><td> </td> <td style="text-align: right">$</td><td id="xdx_98E_eecd--TotalShareholderRtnAmt_pp2d_c20220101__20221231_fKDUp_z7wRx2sj3jT3" style="text-align: right">108</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_98E_eecd--PeerGroupTotalShareholderRtnAmt_pp2d_c20220101__20221231_fKDYp_zByB4Wf9gnFb" style="text-align: right">111</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td style="text-align: right"><span id="xdx_907_eus-gaap--NetIncomeLoss_pn3n3_uUSD_c20220101__20221231_fKDcp_z4eAoovAxFc8">(707,421</span>)</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td style="text-align: right"><span id="xdx_905_eecd--CoSelectedMeasureAmt_pn3n3_uUSD_c20220101__20221231_fKDgp_znFUkpEPa7B5">363,329</span></td><td style="text-align: left"> </td></tr> <tr style="vertical-align: bottom; background-color: White"> <td style="font-weight: bold; text-align: left; padding-left: 3pt">2021</td><td> </td> <td style="text-align: right">$</td><td id="xdx_98C_eecd--PeoTotalCompAmt_c20210101__20211231_fKDEp_zYPy2NOhnS38" style="text-align: right">9,522,738</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_98E_eecd--PeoActuallyPaidCompAmt_c20210101__20211231_fKDIp_z5Lpp8lrjL29" style="text-align: right">(2,152,804)</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_988_eecd--NonPeoNeoAvgTotalCompAmt_c20210101__20211231_fKDMp_z9sv7nnfyuv1" style="text-align: right">3,314,256</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_987_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20210101__20211231_fKDQp_zGyz2uuSUOw1" style="text-align: right">(1,211,638)</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_988_eecd--TotalShareholderRtnAmt_pp2d_c20210101__20211231_fKDUp_z7xSyod7nHa1" style="text-align: right">197</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td id="xdx_982_eecd--PeerGroupTotalShareholderRtnAmt_pp2d_c20210101__20211231_fKDYp_zAcBkgt4oIck" style="text-align: right">125</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td style="text-align: right"><span id="xdx_90D_eus-gaap--NetIncomeLoss_pn3n3_uUSD_c20210101__20211231_fKDcp_zTVhkbr8hA6h">(454,025</span>)</td><td style="text-align: left"> </td><td> </td> <td style="text-align: right">$</td><td style="text-align: right"><span id="xdx_90F_eecd--CoSelectedMeasureAmt_pn3n3_uUSD_c20210101__20211231_fKDgp_zzmdZpz64Wr6">351,406</span></td><td style="text-align: left"> </td></tr> <tr style="vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="border-bottom: rgb(124,188,62) 2px solid; font-weight: bold; text-align: left; padding-left: 3pt">2020</td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_98C_eecd--PeoTotalCompAmt_c20200101__20201231_fKDEp_zyiaAhSKpeXi" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">5,144,220</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_989_eecd--PeoActuallyPaidCompAmt_c20200101__20201231_fKDIp_zBkftAOQQp73" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">22,726,743</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_98D_eecd--NonPeoNeoAvgTotalCompAmt_c20200101__20201231_fKDMp_z490CjBrJMF9" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">2,706,102</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_981_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20200101__20201231_fKDQp_zmGcSNFkvD7l" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">8,015,000</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_98E_eecd--TotalShareholderRtnAmt_pp2d_c20200101__20201231_fKDUp_zoukJa9M5A3d" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">324</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td id="xdx_984_eecd--PeerGroupTotalShareholderRtnAmt_pp2d_c20200101__20201231_fKDYp_zRAMKImZAIui" style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">126</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right"><span id="xdx_90E_eus-gaap--NetIncomeLoss_pn3n3_uUSD_c20200101__20201231_fKDcp_za0ZDSe2bYgg">(186,566</span>)</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right">$</td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: right"><span id="xdx_903_eecd--CoSelectedMeasureAmt_pn3n3_uUSD_c20200101__20201231_fKDgp_znNDdTuyz821">271,030</span></td><td style="border-bottom: rgb(124,188,62) 2px solid; text-align: left"> </td></tr> </table> <p style="margin-top: 0; margin-bottom: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; display: none; width: 100%; border-collapse: collapse; visibility: hidden"> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F08_zUbOcKmjWVi9" style="display: none; width: 18pt; padding-top: 3pt; visibility: hidden">(1)</td> <td id="xdx_F14_zguzDdlyE8A4" style="display: none; text-align: justify; padding-top: 3pt; visibility: hidden">The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for Dr. Kakkis, who served as our Chief Executive Officer during 2020, 2021, 2022 and 2023.</td></tr> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F0C_z3e74aTkUcYf" style="display: none; padding-top: 3pt; visibility: hidden">(2)</td> <td id="xdx_F11_zoiIuBZ4Me2j" style="display: none; text-align: justify; padding-top: 3pt; visibility: hidden">The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP:</td></tr> </table> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; display: none; width: 100%; border-collapse: collapse; visibility: hidden"> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F00_zzudvsMykkh8" style="display: none; width: 18pt; padding-top: 3pt; text-align: left; visibility: hidden">(3)</td> <td id="xdx_F19_zgsuuEWCg0Y6" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (ii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp.</td></tr> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F05_zMR5YaabwLY3" style="display: none; padding-top: 3pt; text-align: left; visibility: hidden">(4)</td> <td id="xdx_F16_zpggjA9p6Eq3" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2:</td></tr> </table> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; display: none; width: 100%; border-collapse: collapse; visibility: hidden"> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F04_zjm8zCQRXIO3" style="display: none; width: 18pt; padding-top: 3pt; text-align: left; visibility: hidden">(5)</td> <td id="xdx_F19_zNAtGU3QVHu9" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">Cumulative TSR is calculated by dividing (a) the sum of (i) the cumulative amount of dividends for the measurement period, assuming dividend reinvestment, and (ii) the difference between the Company’s share price at the end and the beginning of the measurement period by (b) the Company’s share price at the beginning of the measurement period. The beginning of the measurement period for each year in the table is December 31, 2019.</td></tr> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F0A_zZaEFUo1vxh8" style="display: none; padding-top: 3pt; text-align: left; visibility: hidden">(6)</td> <td id="xdx_F13_zEhdIfigS8qf" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index.</td></tr> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F02_zEkNugEZYKOd" style="display: none; padding-top: 3pt; text-align: left; visibility: hidden">(7)</td> <td id="xdx_F15_z2hZ4uEFVvD4" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">The dollar amounts reported represent the amount of net income calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.</td></tr> <tr style="display: none; vertical-align: top; visibility: hidden"> <td id="xdx_F02_zH8dOVffmQBa" style="display: none; padding-top: 3pt; text-align: left; visibility: hidden">(8)</td> <td id="xdx_F13_zeHodNlbVHN7" style="display: none; padding-top: 3pt; text-align: justify; visibility: hidden">The dollar amounts reported represent the amount of revenue calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.</td></tr> </table> <p id="xdx_847_eecd--NamedExecutiveOfficersFnTextBlock_dU_gL2NEOFTB-AB_zVlWlL560QJ4" style="margin-top: 0; margin-bottom: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="width: 18pt; padding-top: 3pt">(1)</td> <td style="text-align: justify; padding-top: 3pt">The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for <span id="xdx_90C_eecd--PeoName_c20230101__20231231_zCGtsbnanbw8"><span id="xdx_902_eecd--PeoName_c20220101__20221231_zXLRWuHHhvyb"><span id="xdx_90F_eecd--PeoName_c20210101__20211231_zBAJiXBNNKl2"><span id="xdx_90C_eecd--PeoName_c20200101__20201231_zmIqbZw05Ovl">Dr. Kakkis</span></span></span></span>, who served as our Chief Executive Officer during 2020, 2021, 2022 and 2023.</td></tr> </table> <p id="xdx_857_zOac3TbxWoea" style="margin: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="padding-top: 3pt; width: 18pt">(2)</td> <td style="text-align: justify; padding-top: 3pt">The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP:</td></tr> </table> <p id="xdx_84C_eecd--AdjToPeoCompFnTextBlock_dU_zdZ5zvjoIWV3" style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <table cellpadding="0" cellspacing="0" id="xdx_30F_zoip6Z2ECnE5" style="font: 8pt Arial, Helvetica, Sans-Serif; border-collapse: collapse; width: 98.5%; margin-left: 18pt" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; padding-left: 3pt">Compensation Actually Paid to PEO</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2023</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2022</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2021</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2020</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 44%; text-align: left; padding-left: 3pt">Summary Compensation Table Total</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_987_eecd--PeoTotalCompAmt_c20230101__20231231_fKDEp_zLcnEgrPY4dd" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">13,077,993</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--PeoTotalCompAmt_c20220101__20221231_fKDEp_zn9qJePLnSil" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">12,140,330</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$  </td><td id="xdx_98B_eecd--PeoTotalCompAmt_c20210101__20211231_fKDEp_zH4MOWY8GAzb" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">9,522,738</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$  </td><td id="xdx_988_eecd--PeoTotalCompAmt_c20200101__20201231_fKDEp_zalpsRCp9kPi" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">5,144,220</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zQV8uAqJYrxg" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(11,618,444)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_z0CnHZWWy4s9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(10,773,976)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zWWXFuh0yAid" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(8,246,092)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98B_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_ziSCKGUCvcY9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,709,589)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus,</i> year end fair value of outstanding and unvested equity awards granted during the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zG45XRpn9JR5" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(6,264,762)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_987_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zcjVNlx4Bmh9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,152,033</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zfT6MxDLoRn7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">10,163,438</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of outstanding and unvested equity awards granted in prior years</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zHwEyd4puHZ6" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,167,400)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_986_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zhn8pJGiIVYa" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,934,552)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zcgGGaVbOOZ" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(5,753,632)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_988_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zqOhYUsAMcw7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">9,346,926</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of equity awards granted in prior years that vested in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zQKMoibVOQl4" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(286,732)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_z92GagrkxOHf" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,385,132)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zGS8XqEq8F44" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,827,851)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_981_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zhbrxlo7g26d" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,781,748</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> prior year-end fair value of any equity awards that failed to meet vesting conditions in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_983_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zqDsM2kNOK3g" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0082">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zNuClxmNmbH8" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0083">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zzjHTeg4q9m7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0084">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_982_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zFRxai2amOch" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0085">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt">Compensation Actually Paid to Dr. Kakkis</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98D_eecd--PeoActuallyPaidCompAmt_c20230101__20231231_fKDEpKDIpKDMp_zTe7YPJ4H8Gi" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">11,293,489</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98A_eecd--PeoActuallyPaidCompAmt_c20220101__20221231_fKDEpKDIpKDMp_zMGvWltq53Sa" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">3,311,432</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$  </td><td id="xdx_981_eecd--PeoActuallyPaidCompAmt_c20210101__20211231_fKDEpKDIpKDMp_zTKTAwz19ZEh" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,152,804)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$  </td><td id="xdx_981_eecd--PeoActuallyPaidCompAmt_c20200101__20201231_fKDEpKDIpKDMp_z7v0jNTOGAGf" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">22,726,743</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> </table> <p id="xdx_855_z9KSta1csVae" style="margin-top: 0; margin-bottom: 0"> </p> <div id="xdx_C04_gL2NEOFTB-AB_zUURweN5iXP8"><p style="margin-top: 0; margin-bottom: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; margin-top: -10pt; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="width: 18pt; padding-top: 3pt; text-align: left">(3)</td> <td style="padding-top: 3pt; text-align: justify">The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (ii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp.</td></tr> </table></div> <p style="margin: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="padding-top: 3pt; text-align: left; width: 18pt">(4)</td> <td style="padding-top: 3pt; text-align: justify">The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2:</td></tr> </table> <div style="border-bottom: Silver 4px solid; margin-bottom: 6pt"></div> <div style="page-break-before: always; margin-top: 6pt; margin-bottom: 10pt"></div> <p id="xdx_840_eecd--AdjToNonPeoNeoCompFnTextBlock_dU_zWvt6FykxGsd" style="margin-top: 0; margin-bottom: 0"> </p> <table cellpadding="0" cellspacing="0" id="xdx_303_zhEUNeBt5KWf" style="font: 8pt Arial, Helvetica, Sans-Serif; border-collapse: collapse; width: 98.5%; margin-left: 18pt" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; padding-left: 3pt">Compensation Actually Paid to Non-PEO NEOs</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2023</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2022</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2021</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2020</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 44%; text-align: left; padding-left: 3pt">Summary Compensation Table Total</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgTotalCompAmt_c20230101__20231231_fKDEp_zzkCv8SftkTb" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">4,524,001</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98B_eecd--NonPeoNeoAvgTotalCompAmt_c20220101__20221231_fKDEp_zozPJgv3C73g" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">3,911,773</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgTotalCompAmt_c20210101__20211231_fKDEp_zWtEr5c7mA1b" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">3,314,256</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98F_eecd--NonPeoNeoAvgTotalCompAmt_c20200101__20201231_fKDEp_zzuHoOjFR8oi" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">2,706,102</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_988_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zpcfe7EyQN0f" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,831,408)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zLYrvCqCWoBg" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,130,468)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_z9ZnzibQb7xd" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,519,639)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zv58OdwOfhsb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,027,088)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus,</i> year end fair value of outstanding and unvested equity awards granted during the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zLKqIzPGu3xi" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,476,201</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_z6zybJ8xqRqj" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,264,910</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zKoI7oTy8nLb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,268,695</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zbXUsNdMQlu5" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,150,071</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of outstanding and unvested equity awards granted in prior years</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98A_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zDIjBB2RCYCc" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(237,537)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_981_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zbhotCVdeosk" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(784,234)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zYZ8LEChACai" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,359,539)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_983_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zES7TeSI4Pd1" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">2,748,610</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of equity awards granted in prior years that vested in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_980_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zyLwReeC9Vyd" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(101,588)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zGfWRanDMcIj" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(573,020)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_986_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zpnK3x5ah6Yh" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(915,411)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zC4ofgptbttb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">437,305</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less, </i>prior year-end fair value of any equity awards that failed to meet vesting conditions in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98A_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zxtUKqHvugSk" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(864,551)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt">Compensation Actually Paid to Non-PEO NEOs</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98A_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20230101__20231231_fKDEpKDIpKDMp_zFR2x834BdAi" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,829,669</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98D_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20220101__20221231_fKDEpKDIpKDMp_ziqg9lck4kvf" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(175,590)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20210101__20211231_fKDEpKDIpKDMp_zljuACi0s1ci" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,211,638)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_984_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20200101__20201231_fKDEpKDIpKDMp_z3LUs86J2gGl" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">8,015,000</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> </table> <p id="xdx_85C_zNtFWRdnEkJj" style="margin-top: 0; margin-bottom: 0"> </p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; margin-top: -10pt; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="width: 18pt; padding-top: 3pt; text-align: left">(5)</td> <td style="padding-top: 3pt; text-align: justify">Cumulative TSR is calculated by dividing (a) the sum of (i) the cumulative amount of dividends for the measurement period, assuming dividend reinvestment, and (ii) the difference between the Company’s share price at the end and the beginning of the measurement period by (b) the Company’s share price at the beginning of the measurement period. The beginning of the measurement period for each year in the table is December 31, 2019.</td></tr> <tr style="vertical-align: top"> <td style="padding-top: 3pt; text-align: left">(6)</td> <td id="xdx_982_eecd--PeerGroupIssuersFnTextBlock_c20230101__20231231_zQBkhUK5hq2i" style="padding-top: 3pt; text-align: justify">Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index.</td></tr> <tr style="vertical-align: top"> <td style="padding-top: 3pt; text-align: left">(7)</td> <td style="padding-top: 3pt; text-align: justify">The dollar amounts reported represent the amount of net income calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.</td></tr> <tr style="vertical-align: top"> <td style="padding-top: 3pt; text-align: left">(8)</td> <td style="padding-top: 3pt; text-align: justify">The dollar amounts reported represent the amount of <span id="xdx_90D_eecd--CoSelectedMeasureName_c20230101__20231231_zZ4WnkXD7rj6">revenue</span> calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.</td></tr> </table> 13077993 11293489 4524001 4829669 112 115 -606639000 434249000 12140330 3311432 3911773 -175590 108 111 -707421000 363329000 9522738 -2152804 3314256 -1211638 197 125 -454025000 351406000 5144220 22726743 2706102 8015000 324 126 -186566000 271030000 <p id="xdx_847_eecd--NamedExecutiveOfficersFnTextBlock_dU_gL2NEOFTB-AB_zVlWlL560QJ4" style="margin-top: 0; margin-bottom: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="width: 18pt; padding-top: 3pt">(1)</td> <td style="text-align: justify; padding-top: 3pt">The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for <span id="xdx_90C_eecd--PeoName_c20230101__20231231_zCGtsbnanbw8"><span id="xdx_902_eecd--PeoName_c20220101__20221231_zXLRWuHHhvyb"><span id="xdx_90F_eecd--PeoName_c20210101__20211231_zBAJiXBNNKl2"><span id="xdx_90C_eecd--PeoName_c20200101__20201231_zmIqbZw05Ovl">Dr. Kakkis</span></span></span></span>, who served as our Chief Executive Officer during 2020, 2021, 2022 and 2023.</td></tr> </table> <p style="margin-top: 0; margin-bottom: 0"></p> <table cellpadding="0" cellspacing="0" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 100%; margin-top: -10pt; border-collapse: collapse"> <tr style="vertical-align: top"> <td style="width: 18pt; padding-top: 3pt; text-align: left">(3)</td> <td style="padding-top: 3pt; text-align: justify">The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (ii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp.</td></tr> </table> Dr. Kakkis Dr. Kakkis Dr. Kakkis Dr. Kakkis <p id="xdx_84C_eecd--AdjToPeoCompFnTextBlock_dU_zdZ5zvjoIWV3" style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <table cellpadding="0" cellspacing="0" id="xdx_30F_zoip6Z2ECnE5" style="font: 8pt Arial, Helvetica, Sans-Serif; border-collapse: collapse; width: 98.5%; margin-left: 18pt" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; padding-left: 3pt">Compensation Actually Paid to PEO</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2023</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2022</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2021</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2020</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 44%; text-align: left; padding-left: 3pt">Summary Compensation Table Total</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_987_eecd--PeoTotalCompAmt_c20230101__20231231_fKDEp_zLcnEgrPY4dd" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">13,077,993</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--PeoTotalCompAmt_c20220101__20221231_fKDEp_zn9qJePLnSil" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">12,140,330</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$  </td><td id="xdx_98B_eecd--PeoTotalCompAmt_c20210101__20211231_fKDEp_zH4MOWY8GAzb" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">9,522,738</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$  </td><td id="xdx_988_eecd--PeoTotalCompAmt_c20200101__20201231_fKDEp_zalpsRCp9kPi" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">5,144,220</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zQV8uAqJYrxg" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(11,618,444)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_z0CnHZWWy4s9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(10,773,976)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zWWXFuh0yAid" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(8,246,092)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98B_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_ziSCKGUCvcY9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,709,589)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus,</i> year end fair value of outstanding and unvested equity awards granted during the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zG45XRpn9JR5" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(6,264,762)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_987_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zcjVNlx4Bmh9" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,152,033</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zfT6MxDLoRn7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">10,163,438</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of outstanding and unvested equity awards granted in prior years</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zHwEyd4puHZ6" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,167,400)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_986_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zhn8pJGiIVYa" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,934,552)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zcgGGaVbOOZ" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(5,753,632)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_988_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zqOhYUsAMcw7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">9,346,926</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of equity awards granted in prior years that vested in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zQKMoibVOQl4" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(286,732)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_z92GagrkxOHf" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,385,132)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zGS8XqEq8F44" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,827,851)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_981_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zhbrxlo7g26d" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,781,748</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> prior year-end fair value of any equity awards that failed to meet vesting conditions in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_983_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zqDsM2kNOK3g" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0082">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zNuClxmNmbH8" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0083">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_984_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zzjHTeg4q9m7" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0084">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_982_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--PeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zFRxai2amOch" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"><span style="-sec-ix-hidden: xdx2ixbrl0085">—</span></td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt">Compensation Actually Paid to Dr. Kakkis</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98D_eecd--PeoActuallyPaidCompAmt_c20230101__20231231_fKDEpKDIpKDMp_zTe7YPJ4H8Gi" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">11,293,489</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98A_eecd--PeoActuallyPaidCompAmt_c20220101__20221231_fKDEpKDIpKDMp_zMGvWltq53Sa" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">3,311,432</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$  </td><td id="xdx_981_eecd--PeoActuallyPaidCompAmt_c20210101__20211231_fKDEpKDIpKDMp_zTKTAwz19ZEh" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,152,804)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$  </td><td id="xdx_981_eecd--PeoActuallyPaidCompAmt_c20200101__20201231_fKDEpKDIpKDMp_z7v0jNTOGAGf" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">22,726,743</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> </table> 13077993 12140330 9522738 5144220 -11618444 -10773976 -8246092 -3709589 -6264762 4152033 10163438 -1167400 -2934552 -5753632 9346926 -286732 -1385132 -1827851 1781748 11293489 3311432 -2152804 22726743 <p id="xdx_840_eecd--AdjToNonPeoNeoCompFnTextBlock_dU_zWvt6FykxGsd" style="margin-top: 0; margin-bottom: 0"> </p> <table cellpadding="0" cellspacing="0" id="xdx_303_zhEUNeBt5KWf" style="font: 8pt Arial, Helvetica, Sans-Serif; border-collapse: collapse; width: 98.5%; margin-left: 18pt" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; padding-left: 3pt">Compensation Actually Paid to Non-PEO NEOs</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2023</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2022</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2021</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td colspan="2" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right">2020</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 44%; text-align: left; padding-left: 3pt">Summary Compensation Table Total</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgTotalCompAmt_c20230101__20231231_fKDEp_zzkCv8SftkTb" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">4,524,001</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98B_eecd--NonPeoNeoAvgTotalCompAmt_c20220101__20221231_fKDEp_zozPJgv3C73g" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">3,911,773</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgTotalCompAmt_c20210101__20211231_fKDEp_zWtEr5c7mA1b" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">3,314,256</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 2%"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 7%; text-align: right; vertical-align: middle">$</td><td id="xdx_98F_eecd--NonPeoNeoAvgTotalCompAmt_c20200101__20201231_fKDEp_zzuHoOjFR8oi" style="font: 8pt Arial, Helvetica, Sans-Serif; width: 4%; text-align: right; vertical-align: middle">2,706,102</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; width: 1%; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less,</i> value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_988_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zpcfe7EyQN0f" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,831,408)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zLYrvCqCWoBg" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(3,130,468)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_z9ZnzibQb7xd" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,519,639)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessValueOfStockAwardsAndOptionAwardsReportedInSummaryCompensationTableMember_zv58OdwOfhsb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,027,088)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"></td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus,</i> year end fair value of outstanding and unvested equity awards granted during the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98C_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zLKqIzPGu3xi" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,476,201</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98F_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_z6zybJ8xqRqj" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,264,910</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zKoI7oTy8nLb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">1,268,695</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusYearEndFairValueOfOutstandingAndUnvestedEquityAwardsGrantedDuringTheYearMember_zbXUsNdMQlu5" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,150,071</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of outstanding and unvested equity awards granted in prior years</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98A_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zDIjBB2RCYCc" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(237,537)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_981_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zbhotCVdeosk" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(784,234)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_989_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zYZ8LEChACai" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(2,359,539)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_983_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfOutstandingAndUnvestedEquityAwardsGrantedInPriorYearsMember_zES7TeSI4Pd1" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">2,748,610</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Plus (less),</i> year over year change in fair value of equity awards granted in prior years that vested in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_980_eecd--AdjToCompAmt_c20230101__20231231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zyLwReeC9Vyd" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(101,588)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98E_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zGfWRanDMcIj" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(573,020)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_986_eecd--AdjToCompAmt_c20210101__20211231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zpnK3x5ah6Yh" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(915,411)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_985_eecd--AdjToCompAmt_c20200101__20201231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--PlusLessYearOverYearChangeInFairValueOfEquityAwardsGrantedInPriorYearsThatVestedInTheYearMember_zC4ofgptbttb" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">437,305</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: rgb(236,244,227)"> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt"><i>Less, </i>prior year-end fair value of any equity awards that failed to meet vesting conditions in the year</td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td id="xdx_98A_eecd--AdjToCompAmt_c20220101__20221231__ecd--ExecutiveCategoryAxis__ecd--NonPeoNeoMember__ecd--AdjToCompAxis__custom--LessPriorYearEndFairValueOfAnyEquityAwardsThatFailedToMeetVestingConditionsInTheYearMember_zxtUKqHvugSk" style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(864,551)</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle"> </td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">—</td><td style="font: 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> <tr style="font: 8pt Arial, Helvetica, Sans-Serif; vertical-align: bottom; background-color: White"> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left; padding-left: 3pt">Compensation Actually Paid to Non-PEO NEOs</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98A_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20230101__20231231_fKDEpKDIpKDMp_zFR2x834BdAi" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">4,829,669</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98D_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20220101__20221231_fKDEpKDIpKDMp_ziqg9lck4kvf" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(175,590)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_98C_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20210101__20211231_fKDEpKDIpKDMp_zljuACi0s1ci" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">(1,211,638)</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif"> </td> <td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">$</td><td id="xdx_984_eecd--NonPeoNeoAvgCompActuallyPaidAmt_c20200101__20201231_fKDEpKDIpKDMp_z3LUs86J2gGl" style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: right; vertical-align: middle">8,015,000</td><td style="border-bottom: rgb(124,188,62) 2px solid; font: bold 8pt Arial, Helvetica, Sans-Serif; text-align: left"> </td></tr> </table> 4524001 3911773 3314256 2706102 -3831408 -3130468 -2519639 -2027088 4476201 1264910 1268695 4150071 -237537 -784234 -2359539 2748610 -101588 -573020 -915411 437305 -864551 4829669 -175590 -1211638 8015000 Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index. revenue <h5 id="xdx_800_eecd--TabularListTableTextBlock_dU_z7gaZQBSQZQ5" style="font: 10pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"><b>Performance Measures</b></h5> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify">As described in greater detail in “Executive Compensation –Compensation Discussion and Analysis,” the Company’s executive compensation program reflects a variable pay-for-performance philosophy. The metrics that the Company uses for both our long-term and short-term incentive awards are selected based on an objective of incentivizing our NEOs to increase the value of our enterprise for our shareholders. The most important performance measures used by the Company to link executive compensation actually paid to the Company’s NEOs, for the most recently completed fiscal year, to the Company’s performance are as follows:</p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify"> </p> <table cellpadding="0" cellspacing="0" id="xdx_305_zjXrYNF2pMe1" style="font: 9pt Arial, Helvetica, Sans-Serif; width: 100%; border-collapse: collapse" summary="xdx: Disclosure - Pay vs Performance Disclosure"> <tr style="vertical-align: top"> <td style="width: 10pt; padding-bottom: 5pt"><span style="color: #7CBC3E">•</span></td> <td style="padding-bottom: 5pt"><span id="xdx_90C_eecd--MeasureName_c20230101__20231231__ecd--MeasureAxis__1_zbutbTGHqg8">Total Revenue</span> </td></tr> <tr style="vertical-align: top"> <td style="padding-bottom: 5pt"><span style="color: #7CBC3E">•</span></td> <td style="padding-bottom: 5pt"><span id="xdx_902_eecd--MeasureName_c20230101__20231231__ecd--MeasureAxis__2_zQnYyfI0DNY2">Relative Total Stockholder Return</span> </td></tr> <tr style="vertical-align: top"> <td style="padding-bottom: 5pt"><span style="color: #7CBC3E">•</span></td> <td style="padding-bottom: 5pt"><span id="xdx_907_eecd--MeasureName_c20230101__20231231__ecd--MeasureAxis__3_zayCTZjyGmrh">Net Cash Used in Operations </span></td></tr> <tr style="vertical-align: top"> <td><span style="color: #7CBC3E">•</span></td> <td><span id="xdx_90D_eecd--MeasureName_c20230101__20231231__ecd--MeasureAxis__4_zDxt7j0E05e2">Clinical Development of Product Candidates</span></td></tr> </table> Total Revenue Relative Total Stockholder Return Net Cash Used in Operations Clinical Development of Product Candidates <p id="xdx_800_eecd--CompActuallyPaidVsTotalShareholderRtnTextBlock_dU_zmnJrL0LihNl" style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: justify">The following graphs provide visual representations of the relationship between both the CAP of our PEO and the average CAP of our non-PEO NEOs and our (i) TSR, (ii) net income, and (iii) Revenue.</p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: center"> </p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin-top: 0; margin-bottom: 0; text-align: center"><img alt="" src="lrarex71x1.jpg"/></p> <p id="xdx_809_eecd--CompActuallyPaidVsNetIncomeTextBlock_dU_zuKOxMpHzNUl" style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: center"> </p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin-top: 0; margin-bottom: 0; text-align: center"><img alt="" src="lrarex71x2.jpg"/></p> <p id="xdx_808_eecd--CompActuallyPaidVsCoSelectedMeasureTextBlock_dU_zILa43ZRfAZ4" style="font: 9pt Arial, Helvetica, Sans-Serif; margin: 0; text-align: center"> </p> <p style="font: 9pt Arial, Helvetica, Sans-Serif; margin-top: 0; margin-bottom: 0; text-align: center"><img alt="" src="lrarex71x3.jpg"/></p> The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for Dr. Kakkis, who served as our Chief Executive Officer during 2020, 2021, 2022 and 2023. The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP: The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2023, Mr. Horn, Mr. Harris, Mr. Pinion and Ms. Parschauer, (ii) for 2022 and 2021, Dr. Camille Bedrosian, Mr. Harris, Mr. Pinion and Mardi Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp. The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2: Cumulative TSR is calculated by dividing (a) the sum of (i) the cumulative amount of dividends for the measurement period, assuming dividend reinvestment, and (ii) the difference between the Company’s share price at the end and the beginning of the measurement period by (b) the Company’s share price at the beginning of the measurement period. The beginning of the measurement period for each year in the table is December 31, 2019. Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index. The dollar amounts reported represent the amount of net income calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year. The dollar amounts reported represent the amount of revenue calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.