100000 - Document - Document and Entity Information
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100010 - Statement - CONSOLIDATED BALANCE SHEETS
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100020 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details) 2
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100030 - Statement - CONSOLIDATED BALANCE SHEETS (Parenthetical)
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100040 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details) 3
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100050 - Statement - CONSOLIDATED STATEMENTS OF OPERATIONS
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100060 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details) 4
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100070 - Statement - CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS
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100080 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details) 5
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100090 - Statement - CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY
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100100 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details) 6
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100110 - Statement - CONSOLIDATED STATEMENTS OF CASH FLOWS
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995455 - Disclosure - Organization and Basis of Presentation
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995465 - Disclosure - Summary of Significant Accounting Policies
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995475 - Disclosure - Fair Value Measurements
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995485 - Disclosure - Balance Sheet Components
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995495 - Disclosure - Intangible Assets, Net
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995505 - Disclosure - Revenue
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995515 - Disclosure - GeneTx Acquisition
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995525 - Disclosure - License and Research Agreements
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995535 - Disclosure - Leases
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995545 - Disclosure - Liabilities for Sales of Future Royalties
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995555 - Disclosure - Equity
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995565 - Disclosure - Stock-Based Awards
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995575 - Disclosure - Defined Contribution Plan
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995585 - Disclosure - Income Taxes
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995595 - Disclosure - Commitments and Contingencies
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995605 - Disclosure - Related Party Transaction
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995615 - Disclosure - Net Loss per Share
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995625 - Disclosure - Accumulated Other Comprehensive Income (Loss)
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995635 - Disclosure - Subsequent Event
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995645 - Disclosure - Summary of Significant Accounting Policies (Policies)
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995655 - Disclosure - Summary of Significant Accounting Policies (Tables)
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995665 - Disclosure - Fair Value Measurements (Tables)
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995675 - Disclosure - Balance Sheet Components (Tables)
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995685 - Disclosure - Intangible Assets, net (Table)
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995695 - Disclosure - Revenue (Tables)
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995705 - Disclosure - License and Research Agreements (Tables)
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995715 - Disclosure - Leases (Tables)
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995725 - Disclosure - Liabilities for Sales of Future Royalties (Tables)
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995735 - Disclosure - Stock-Based Awards (Tables)
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995745 - Disclosure - Income Taxes (Tables)
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995755 - Disclosure - Commitments and Contingencies (Tables)
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995765 - Disclosure - Net Loss per Share (Tables)
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995775 - Disclosure - Accumulated Other Comprehensive Income (Loss) (Tables)
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995785 - Disclosure - Organization and Basis of Presentation - Additional Information (Details)
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995795 - Disclosure - Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash (Details)
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995805 - Disclosure - Summary of Significant Accounting Policies - Additional Information (Details)
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995815 - Disclosure - Fair Value Measurements - Summary of Financial Assets Measured on Recurring Basis (Details)
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995825 - Disclosure - Fair Value Measurements - Additional Information (Details)
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995835 - Disclosure - Balance Sheet Components - Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities (Details)
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995845 - Disclosure - Balance Sheet Components - Additional Information (Details)
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995855 - Disclosure - Balance Sheet Components - Summary of Inventory (Details)
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995865 - Disclosure - Balance Sheet Components - Summary of Property, Plant and Equipment, Net (Details)
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995875 - Disclosure - Balance Sheet Components - Summary of Accrued Liabilities (Details)
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995885 - Disclosure - Intangible Assets, Net - Additional Information (Details) 1
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995895 - Disclosure - Intangible Assets, Net - Additional Information (Details)
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995905 - Disclosure - Intangible Assets, Net - Summary of Company's Intangible Assets (Details)
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995915 - Disclosure - Intangible Assets, Net - Schedule of Expected Amortization of the Intangible Assets (Details)
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995925 - Disclosure - Revenue - Summary of Disaggregation of Total Revenues from Customers (Details)
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995935 - Disclosure - Revenue - Summary of Disaggregation of Total Revenues (Details)
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995945 - Disclosure - Revenue - Schedule of Sales-Related Accruals and Allowances (Details)
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995955 - Disclosure - Revenue - Summary of Changes in Contract Liabilities (Details)
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995965 - Disclosure - Revenue - Additional Information (Details)
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995975 - Disclosure - GeneTx Acquisition - Additional Information (Details)
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995985 - Disclosure - License and Research Agreements - Kyowa Kirin Co., Ltd. - Additional Information (Details)
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995995 - Disclosure - License and Research Agreements - Saint Louis University - Additional Information (Details)
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996005 - Disclosure - License and Research Agreements - Baylor Research Institute - Additional Information (Details)
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996015 - Disclosure - License and Research Agreements - REGENXBIO, Inc. - Additional Information (Details)
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996025 - Disclosure - License and Research Agreements - University of Pennsylvania - Additional Information (Details)
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996035 - Disclosure - License and Research Agreements - Arcturus Therapeutics Holdings Inc. - Additional Information (Details)
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996045 - Disclosure - License and Research Agreements - Daiichi Sankyo - Additional Information (Details)
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996055 - Disclosure - License and Research Agreements - Mereo - Additional Information (Details)
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996065 - Disclosure - License and Research Agreements - Regeneron - Additional Information (Details)
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996075 - Disclosure - License and Research Agreements - Abeona - Additional Information (Details)
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996085 - Disclosure - License and Research Agreements - Solid Biosciences, Inc. - Additional Information (Details)
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996095 - Disclosure - License and Research Agreements - Share of Collaboration and Royalty Revenue Related to Crysvita (Details)
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996105 - Disclosure - License and Research Agreements - Schedule of Collaboration Cost Sharing and Payments (Details)
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996115 - Disclosure - License and Research Agreements - Schedule of Changes in Fair Value of Equity Investment (Details)
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996125 - Disclosure - License and Research Agreements - Schedule of Changes in Fair Value of Investment in Securities (Details)
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996135 - Disclosure - Leases - Additional Information (Details)
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996145 - Disclosure - Leases - Summary of Components of Lease Expense (Details)
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996155 - Disclosure - Leases - Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities (Details)
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996165 - Disclosure - Liabilities for Sales of Future Royalties - Additional Information (Details)
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996175 - Disclosure - Liabilities for Sales of Future Royalties - Schedule of Activity Within Liability Related to Sale of Future Royalties (Details)
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996185 - Disclosure - Equity - Additional Information (Details)
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996195 - Disclosure - Stock-Based Awards - Additional Information (Details)
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996205 - Disclosure - Stock-Based Awards - Summary of Activity Under Stock Option Plans Including 2011 Plan and 2014 Plan and 2023 Plan (Details)
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996215 - Disclosure - Stock-Based Awards- Summary Activity Under the Performance Stock Option (Details)
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996225 - Disclosure - Stock-Based Awards - Summary of Activity Under Restricted Stock Units (RSUs) from 2014 Plan (Details)
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996235 - Disclosure - Stock-Based Awards - Summary of Activity Under PSUs from 2014 Plan (Details)
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996245 - Disclosure - Stock-Based Awards - Summary of Stock-Based Compensation Recognized (Details)
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996255 - Disclosure - Stock-Based Awards - Fair Value of Stock Option Awards Granted Estimated Using Black-Scholes Option-Pricing Model (Details)
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996265 - Disclosure - Defined Contribution Plan - Additional Information (Details)
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996275 - Disclosure - Income Taxes - Components of Company's Loss Before Income Taxes (Details)
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996285 - Disclosure - Income Taxes - Components of Company's Income Tax Provision (Details)
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996295 - Disclosure - Income Taxes - Additional Information (Details)
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996305 - Disclosure - Income Taxes - Effective Tax Rate of Provision for Income Taxes from Federal Statutory Rate (Details)
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996315 - Disclosure - Income Taxes - Schedule of Tax Effect of Temporary Differences to Significant Portions of Deferred Tax Assets (Details)
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996325 - Disclosure - Income Taxes - Unrecognized Tax Benefits (Details)
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996335 - Disclosure - Commitments and Contingencies - Additional Information (Details)
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996345 - Disclosure - Commitments and Contingencies - Summary of Aggregate Payments under Contractually Binding Manufacturing and Service Agreements (Details)
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996355 - Disclosure - Related Party Transaction - Additional Information (Details)
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996365 - Disclosure - Net Loss per Share - Computation of Basic and Diluted Net Loss per Share (Details)
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996375 - Disclosure - Net Loss per Share - Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share (Details)
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996385 - Disclosure - Accumulated Other Comprehensive Income (Loss) - Schedule of Total Accumulated Other Comprehensive Income (Loss) (Details)
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Upfornt cash payment paid
Upfornt cash payment paid.
Auditor Firm ID
2024
Lessee, Operating Lease, Liability, to be Paid, Year One
Collaborative Arrangement and Arrangement Other than Collaborative
Collaborative Arrangement and Arrangement Other than Collaborative [Domain]
Document Transition Report
Document Transition Report
Expected volatility
Share-Based Compensation Arrangement by Share-Based Payment Award, Fair Value Assumptions, Expected Volatility Rate
Weighted Average Remaining Contractual Term (Years), Outstanding
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Remaining Contractual Term
Deferred Tax Assets, in Process Research and Development
Capitalized research and development costs
Money Market Funds
Money Market Funds [Member]
Development And Transition Costs
Development And Transition Costs
Development and transition costs
Effective Income Tax Rate Reconciliation, Other Reconciling Items, Percent, Total
Other
Effective Income Tax Rate Reconciliation, Other Reconciling Items, Percent
Profit Share Revenue
Profit Share Revenue And Royalties [Member]
Profit share revenue and royalties.
Geographical
Geographical [Axis]
Undistributed earnings of foreign subsidiaries
Undistributed Earnings of Foreign Subsidiaries
Stock based awards vesting period
Share-Based Compensation Arrangement by Share-Based Payment Award, Award Vesting Period
Estimated Fair Value
Debt Securities, Available-for-Sale
Debt Securities, Available-for-sale, Total
Other Current Assets
Other Current Assets [Member]
Finance Lease, Liability, Current, Statement of Financial Position [Extensible Enumeration]
Other liabilities
Other Liabilities, Noncurrent
Other Liabilities, Noncurrent, Total
Texas A&M University [Member]
Texas A&M University [Member]
Texas A&M University [Member]
Number of warrants exercised
Class of Warrant or Right Warrants Exercised
Class of warrant or right, warrants exercised.
Restricted Stock Units
Restricted Stock Units (RSUs) [Member]
2025
Finite-Lived Intangible Asset, Expected Amortization, Year Two
Level 1
Fair Value, Inputs, Level 1 [Member]
Entity Public Float
Entity Public Float
Short-Term Debt [Member]
Deferred Compensation Obligation
Deferred Compensation, Share-Based Payments [Member]
2027
Lessee, Operating Lease, Liability, to be Paid, Year Four
Depreciation expense
Depreciation Excluding Intangible Amortization
Depreciation excluding intangible amortization.
Fair Value Measurements
Fair Value Disclosures [Text Block]
Total future lease payments
Finance Lease, Liability, to be Paid
Income Taxes
Income Tax, Policy [Policy Text Block]
Property, plant and equipment, estimated useful lives (years)
Property plant and equipment useful life
Property, Plant and Equipment, Useful Life
Revenue
Revenue from Contract with Customer, Excluding Assessed Tax, Total
Collaboration and license revenue
Total revenues
Schedule of Activity Within Liability for Sale of Future Royalties
Schedule of activity within liability for sale of future royalties.
Schedule Of Activity Within Liability For Sale Of Future Royalties [Table Text Block]
License Agreement
License Agreement [Member]
License agreement.
Pre Funded Warrant
Pre Funded Warrant [Member]
Pre Funded Warrant [Member]
Common stock, Sale of shares
Fair Value Of Sale Of Shares
Fair value of sale of shares.
Customer One
Customer One [Member]
Customer One.
Valuation allowance increased
Valuation Allowance, Deferred Tax Asset, Increase (Decrease), Amount
Contract liabilities, net
Increase Decrease In Contract Liabilities
Increase decrease in contract liabilities.
Summary of Disaggregation of Total Revenues
Disaggregation of Revenue [Table Text Block]
Payments to Acquire Businesses, Net of Cash Acquired, Total
Payments to Acquire Businesses, Net of Cash Acquired
Acquisition, net of cash acquired
Acquisition, net of cash acquired
Short-term Debt, Type
Short-Term Debt, Type [Axis]
ICFR Auditor Attestation Flag
ICFR Auditor Attestation Flag
Long-Term Debt [Member]
Organization and Basis of Presentation
Organization, Consolidation, Basis of Presentation, Business Description and Accounting Policies [Text Block]
Schedule of Business Acquisitions, by Acquisition [Table]
Common Stock Warrants Expiring on 2020
Common Stock Warrants Expiring On Two Thousand Twenty [Member]
Common stock warrants expiring on two thousand twenty.
Additional ATM Sales Agreement
Additional At The Market Sales Agreement [Member]
Additional at-the-market sales agreement.
Subsequent Event
Subsequent Events [Text Block]
Operating Lease, Liability, Noncurrent
Lease liabilities, non-current
Corsee Sanders [Member]
Corsee Sanders [Member]
Corsee Sanders.
Investments, Fair Value Disclosure, Total
Total fair value
Common stock, Beginning balance
Common stock, Ending balance
Investments, Fair Value Disclosure
Europe [Member]
Europe
Debt Securities Available For Sale [Table]
Debt Securities, Available-for-Sale [Table]
Lease liabilities, non-current
Finance Lease, Liability, Noncurrent
Time Deposits
Time Deposits [Member]
Time deposits.
Related Party Transactions [Abstract]
Balance Sheet Location
Balance Sheet Location [Axis]
Total current assets
Assets, Current
Vesting
Vesting [Domain]
Extinguishment of Debt, Type [Domain]
Deferred Revenue Recognized
Deferred Revenue, Revenue Recognized
Total future lease payments
Operating And Finance Lease Liability Payments Due
Operating and finance lease liability payments due.
Total liabilities and stockholders’ equity
Liabilities and Equity
Commitments And Contingencies Disclosure Line Items
Commitments And Contingencies Disclosure [Line Items]
Commitments and contingencies disclosure.
Entity Address, State or Province
Entity Address, State or Province
Other
Deferred Tax Assets, Other
Deferred tax provision:
Deferred Income Tax Expense (Benefit), Continuing Operations [Abstract]
2011 Equity Incentive Plan
Two Thousand Eleven Equity Incentive Plan [Member]
Two thousand eleven equity incentive plan.
Stockholders' Equity Note, Stock Split, Conversion Ratio
stock split conversion ratio
Number of Options, Outstanding, Beginning Balance
Number of Options, Outstanding, Ending Balance
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Number
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Grants in Period, Weighted Average Grant Date Fair Value
weighted-average estimated fair value
Total comprehensive loss
Comprehensive Income (Loss), Net of Tax, Attributable to Parent
Non-current lease liability
Lease liabilities
Operating And Finance Lease Liability Noncurrent
Operating and finance lease liability noncurrent.
Trading Symbol
Trading Symbol
Total deferred tax assets
Deferred Tax Assets, Net of Valuation Allowance
Common Stock, Shares, Issued, Total
Common stock purchased
Common stock, shares issued
Common Stock, Shares, Issued
Stock-based compensation
Effective Income Tax Rate Reconciliation, Nondeductible Expense, Share-Based Payment Arrangement, Percent
Schedule Of Available For Sale Securities [Line Items]
Debt Securities, Available-for-Sale [Line Items]
Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash
Restrictions on Cash and Cash Equivalents [Table Text Block]
Total fair value of shares vested
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Vested in Period, Fair Value
University of Pennsylvania School of Medicine (Penn)
University Of Pennsylvania School Of Medicine [Member]
University of Pennsylvania school of medicine.
Defined Contribution Plan
Compensation and Employee Benefit Plans [Text Block]
Equity Investments
Equity Method Investments [Policy Text Block]
Finance Lease, Liability, Noncurrent, Statement of Financial Position [Extensible Enumeration]
Option and License Agreement
Option And License Agreement [Member]
Option and license agreement.
Accounts Receivable, after Allowance for Credit Loss, Total
License agreement accounts receivable
Accounts Receivable, after Allowance for Credit Loss
Contract liabilities
Contract Liabilities Noncurrent
Contract liabilities noncurrent.
Investments in Arcturus and Solid Common Stock
Investments In Arcturus And Solid Common Stock [Member]
Investments in Arcturus and solid common stock.
Non Cash interest expense on liability for sale of future royalties and capitalized to property plant and equipment.
Non Cash Interest Expense On Liability For Sale Of Future Royalties And Capitalized To Property Plant And Equipment
Non-cash interest expense
2026
Lessee, Operating Lease, Liability, to be Paid, Year Three
Deferred compensation assets
Deferred Compensation Assets [Member]
Deferred compensation assets.
Accumulated Other Comprehensive Income (Loss)
AOCI Attributable to Parent [Member]
Business Combination Disclosure [Text Block]
Acquisition
Other comprehensive income (loss):
Other Comprehensive Income (Loss), Net of Tax
Other comprehensive income (loss)
Foreign Currency
Foreign Currency Transactions and Translations Policy [Policy Text Block]
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax, shares
Issuance Of Common Stock Upon Exercise Of Warrants And Under Equity Plan Awards Net Of Tax Shares
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax, shares.
Entity Address, City or Town
Entity Address, City or Town
Related Party Transaction Line Items
Related Party Transaction [Line Items]
Percentage of gross accounts receivable balance
Percentage Of Gross Accounts Receivable Balance
Percentage of gross accounts receivable balance.
Operating Lease, Liability, Statement of Financial Position [Extensible List]
Operating Lease, Liability, Noncurrent, Statement of Financial Position [Extensible Enumeration]
Operating lease weighted average discount rate
Operating Lease, Weighted Average Discount Rate, Percent
Common stock warrants outstanding
Class of Warrant or Right, Outstanding
Automatic increases in shares available for grant effective date
Share Based Compensation Arrangement By Share Based Payment Award Shares Available For Grant Beginning Date
Share based compensation arrangement by share based payment award shares available for grant beginning date.
Summary of Property, Plant and Equipment, Net
Property, Plant and Equipment [Table Text Block]
Deferred tax assets operating loss carryforwards subject to expiration
Deferred Tax Assets, Operating Loss Carryforwards, Subject to Expiration
Additions for tax positions of prior years
Unrecognized Tax Benefits, Increase Resulting from Prior Period Tax Positions
Option extension payment.
Option Extension Payment
Option extension payment
2025
Operating And Finance Lease Liability Payments Due Year Two
Operating and finance lease liability payments due year two.
Additional Paid-in Capital
Additional Paid-in Capital [Member]
Fair Value of Stock Option Awards Granted Estimated Using Black-Scholes Option-Pricing Model
Schedule Of Share Based Payment Award Stock Options And Employee Stock Purchase Plan Valuation Assumptions Table [Text Block]
Schedule of share based payment award stock options and employee stock purchase plan valuation assumptions.
Components of Company's Income Tax Provision
Schedule of Components of Income Tax Expense (Benefit) [Table Text Block]
Collaboration and license
Collaboration And License [Member]
Collaboration and license.
Property, plant and equipment, estimated useful lives
Property, Plant, and Equipment, Useful Life, Term, Description [Extensible Enumeration]
Proceeds from maturities of marketable debt securities
Proceeds from Maturities, Prepayments and Calls of Debt Securities, Available-for-Sale
Current liabilities:
Liabilities, Current [Abstract]
Current assets:
Assets, Current [Abstract]
Counterparty Name
Counterparty Name [Axis]
Contingent milestone payment
Contingent Milestone Payment
Contingent milestone payment.
Preferred stock, par value
Preferred Stock, Par or Stated Value Per Share
Acquired lease liabilities arising from obtaining right-of-use assets and property, plant, and equipment
Operating And Finance Lease Liability From Obtaining Right Of Use Assets And Property Plant And Equipment
Operating and finance lease liability from obtaining right of use assets and property plant and equipment.
Statement of Stockholders' Equity [Abstract]
2027
Operating And Finance Lease Liability Payments Due Year Four
Operating and finance lease liability payments due year four.
Intangible Assets, Net
Intangible Assets Disclosure [Text Block]
Weighted Average Exercise Price, Vested and expected to vest December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested and Expected to Vest, Outstanding, Weighted Average Exercise Price
Less: Lease liabilities, current
Operating Lease, Liability, Current
Property, Plant and Equipment, Net, Ending Balance
Property, Plant and Equipment, Net, Beginning Balance
Property, plant, and equipment, net
Property, Plant and Equipment, Net
Property, plant and equipment, net
Deferred tax liability increase (decrease), net
Increase (Decrease) in Deferred Income Taxes
Interest income
Investment Income, Interest
Stockholders' Equity Note, Stock Split
Description of the stock split
Adjustments
Sales Reserves Adjustments
Sales reserves adjustments.
2014 Incentive Plan
Two Thousand Fourteen Incentive Plan [Member]
2014 Incentive plan.
Business combination recognized intangible assets
Business Combination, Recognized Identifiable Assets Acquired and Liabilities Assumed, Indefinite-Lived Intangible Assets
Deferred Tax Assets Liability Related To Sale Of Future Royalties
Deferred tax assets liability related to sale of future royalties.
Liabilities for sales of future royalties
Common stock warrants, expiration period
Class Of Warrant Or Right Expiration Year
Class of warrant or right expiration year.
Aggregate Intrinsic Value, Vested and expected to vest - December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested and Expected to Vest, Outstanding, Aggregate Intrinsic Value
Federal statutory income tax rate
Effective Income Tax Rate Reconciliation, at Federal Statutory Income Tax Rate, Percent
Statement of Comprehensive Income [Abstract]
Collaborative Arrangement and Arrangement Other than Collaborative
Collaborative Arrangement and Arrangement Other than Collaborative [Axis]
Treasury stock , shares
Treasury Stock, Common, Shares
Entity Central Index Key
Entity Central Index Key
Less: Lease liabilities, current
Finance Lease, Liability, Current
Plan Name
Plan Name [Domain]
Annual maintenance fee for each option exercised
Licensed Option Maintenance Fee
Licensed option maintenance fee.
Commercial and Development Activity Reimbursements
Commercial And Development Activity Reimbursements [Member]
Commercial and development activity reimbursements.
Weighted-Average Remaining Contractual Term,Outstanding at December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Outstanding, Weighted Average Remaining Contractual Terms
Restricted cash included in other non-current assets
Restricted Cash, Noncurrent
Business acquisition option exercise period, description
Exclusive Options Subject To Option Exercise Period
Exclusive options subject to option exercise period.
Other assets
Increase (Decrease) in Other Operating Assets
Increase (Decrease) in Other Operating Assets, Total
Operating lease extension details
Lessee, Operating Lease, Option to Extend
Preferred Stock, Shares Outstanding, Ending Balance
Preferred Stock, Shares Outstanding, Beginning Balance
Preferred stock, shares outstanding
Preferred Stock, Shares Outstanding
Royalties transaction costs net
Royalties Transaction Costs Net
Royalties transaction costs net.
Total current liabilities
Liabilities, Current
Entity Tax Identification Number
Entity Tax Identification Number
Intangible Assets
Goodwill and Intangible Assets, Intangible Assets, Policy [Policy Text Block]
Total loss before income taxes
Loss before income taxes
Income (Loss) from Continuing Operations before Income Taxes, Noncontrolling Interest
Operating Lease, Right-of-Use Asset, Statement of Financial Position [Extensible Enumeration]
Certificates of Deposit and Time Deposits
Certificate Of Deposits And Time Deposits [Member]
Certificate of deposits and time deposits.
Schedule Of Antidilutive Securities Excluded From Computation Of Earnings Per Share [Table]
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share [Table]
Changes in operating assets and liabilities:
Increase (Decrease) in Operating Capital [Abstract]
Basis difference in equity investments
Deferred Tax Assets, Equity Method Investments
2027
Finance Lease, Liability, to be Paid, Year Four
Financial Instruments [Domain]
Debt instrument maturity period
Debt Instrument, Term
Additional option to purchase common stock
Option to purchase additional common stock, shares
Option To Purchase Additional Common Stock
Option to purchase additional common stock.
Construction-in-progress
Construction in Progress [Member]
Recognized accrued interest and penalties
Income Tax Examination, Penalties and Interest Accrued
Income Tax Examination, Penalties and Interest Accrued, Total
Leases
Lessee, Operating Leases [Text Block]
Financing lease weighted average remaining lease term
Finance Lease, Weighted Average Remaining Lease Term
Right-of-use Lease Assets
Right Of Use Assets [Member]
Right-of-use assets.
Share-Based Payment Arrangement, Disclosure [Abstract]
Plan Name
Plan Name [Axis]
Option and license agreement description
Collaborative Arrangement, Nature and Purpose
Geographical
Geographical [Domain]
Upfront payment received
Revenue from Collaborative Arrangement, Excluding Revenue from Contract with Customer
Total assets
Assets
Remaining consideration paid
Remaining Business Combination Consideration Transferred1
Remaining business combination consideration transferred1.
Issuance of common stock under equity plan awards, net of tax
Issuance Of Common Stock Under Equity Plan Awards Net Of Tax
Issuance of common stock under equity plan awards, net of tax.
Right-of-use lease assets
Operating Lease, Right-of-Use Asset
Matthew Fust [Member]
Matthew Fust [Member]
Matthew Fust.
Total unrecognized compensation cost
Share-Based Payment Arrangement, Nonvested Award, Option, Cost Not yet Recognized, Amount
Commercial milestone payment.
Commercial Milestone Payment
Commercial milestone payment
Royalty payment termination description
Royalty Agreement Termination Description
Royalty agreement termination description.
Entity Registrant Name
Entity Registrant Name
Asset-backed Securities
Asset-Backed Securities [Member]
Granted.
Granted [Member]
Operating lease, existence of option to extend
Lessee, Operating Lease, Existence of Option to Extend [true false]
Related Party
Related Party, Type [Domain]
Retained Earnings (Accumulated Deficit), Ending Balance
Retained Earnings (Accumulated Deficit), Beginning Balance
Retained Earnings (Accumulated Deficit), Total
Adjustment to retained earnings
Accumulated deficit
Retained Earnings (Accumulated Deficit)
Non Profit Foundation [Member]
Non Profit Foundation [Member]
Non Profit Foundation
Accumulated Deficit
Retained Earnings [Member]
Stockholders Equity [Table]
Stockholders Equity [Table]
Stockholders equity table.
Common stock, Acquisition of equity investment
Acquisition Of Equity Investment In Common Stock
Acquisition of equity investment in common stock.
Outstanding, Number of Shares, Unvested, Beginning Balance
Outstanding, Number of Shares, Unvested, Ending Balance
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Number
Federal Income Tax Expense (Benefit), Continuing Operations, Total
Federal
Federal Income Tax Expense (Benefit), Continuing Operations
Deferred Income Tax Liabilities, Net, Total
Deferred tax liabilities
Deferred Income Tax Liabilities, Net
Minimum
Minimum [Member]
Cash received from exercise of options
Proceeds from Stock Options Exercised
Summary of Significant Accounting Policies
Significant Accounting Policies [Text Block]
Crysvita
Crysvita [Member]
Crysvita.
Operating lease expiry term
Lessee Operating Lease Expiry Year
Lessee operating lease expiry year.
Present value of future lease payments
Operating Lease, Liability
Less: Lease liabilities, current
Current lease liability
Lease liabilities
Operating And Finance Lease Liability Current
Operating and finance lease liability current.
Issuance of common stock in connection with license agreement, net of issuance costs, shares
Issuance Of Common Stock In Connection With License Agreement Net Of Issuance Costs Shares
Issuance of common stock in connection with license agreement net of issuance costs shares.
Present value of future lease payments
Finance Lease, Liability
Revenue Recognition
Revenue [Policy Text Block]
Disaggregation of Revenue [Abstract]
Equity Component
Equity Component [Domain]
Defined Contribution Plan, Cost
Defined Contribution Plan Cost Recognized
Contribution expenses
Stock Options
Development Milestones
Development Milestone [Member]
Development milestone.
Land
Land [Member]
Finite-Lived Intangible Assets [Member]
Finite lived intangible assets
Definite-Lived Intangible Asset
Upfront payment for in-process research and development expense
Upfront Payment For In Process Research And Development Expense
Upfront payment for in process research and development expense.
Common Stock Warrants Expiring on 2021
Common Stock Warrants Expiring On Two Thousand Twenty One [Member]
Common stock warrants expiring on two thousand twenty one.
Adjustments to reconcile net loss to net cash used in operating activities:
Adjustments to Reconcile Net Income (Loss) to Cash Provided by (Used in) Operating Activities [Abstract]
Issuance of common stock in connection with at-the-market offering, net of issuance costs
Stock Issued During Period Value At Market Offering
Stock issued during period value at market offering.
Entity Current Reporting Status
Entity Current Reporting Status
Other
Proceeds from (Payments for) Other Financing Activities
Common stock, shares authorized
Common Stock, Shares Authorized
Research and Development Expense, Total
Research and development expense related to amortization of assets
Research and development
Research and Development Expense
Remaining profit or loss share percentage on commercializing products
Profit Loss Sharing Percentage On Commercializing Products
Profit loss sharing percentage on commercializing products.
Other
Other Capitalized Property Plant and Equipment [Member]
Deferred Tax Liabilities, Gross, Total
Gross deferred tax liabilities
Deferred Tax Liabilities, Gross
International
Deferred Foreign Income Tax Expense (Benefit)
International
ASSETS
Assets [Abstract]
Net proceeds from sale of common stock
Proceeds from the issuance of common stock in connection with underwritten public offerings, net
Proceeds from Issuance of Common Stock
Goodwill
Goodwill and Intangible Assets, Goodwill, Policy [Policy Text Block]
Amortization of Intangible Assets
Costs of sales related to amortization of intangible asset
Share of common stock available for grant
Share-Based Compensation Arrangement by Share-Based Payment Award, Number of Shares Available for Grant
Open Market Sale
Open Market Sale [Member]
Open market sale.
License and collaboration agreement.
License And Collaboration Agreement [Member]
License and Collaboration Agreement
Common stock, par value of $0.001 per share - 250,000,000 shares authorized;outstanding - 82,315,590 in 2023 and 70,197,297 in 2022
Common Stock, Value, Issued, Ending Balance
Common Stock, Value, Issued, Beginning Balance
Common Stock, Value, Issued, Total
Common stock investment value
Common Stock, Value, Issued
Change in valuation allowance
Effective Income Tax Rate Reconciliation, Change in Deferred Tax Assets Valuation Allowance, Percent
Other Noncash Income (Expense), Total
Other
Other Noncash Income (Expense)
Dojolvi
Dojolvi [Member]
Dojolvi.
Revenue recognized
Contract with Customer, Liability, Revenue Recognized
Accounts payable, accrued, and other liabilities
Increase Decrease In Accounts Payable Accrued Liabilities And Other Operating Liabilities
Increase decrease in accounts payable accrued liabilities and other operating liabilities.
Total accrued liabilities
Accrued liabilities
Accrued Liabilities, Current
Current Fiscal Year End Date
Current Fiscal Year End Date
Auditor Name
Loss from operations
Operating Income (Loss)
Business Combination, Consideration Transferred, Total
Business acquisition, consideration transferred
Business Combination, Consideration Transferred
Total deferred tax provision
Deferred income tax expense
Total deferred tax provision
Deferred Income Tax Expense (Benefit)
Cost Sharing Payments [Line Items]
Cost Sharing Payments [Line Items]
Cost sharing payments line items.
Total (benefit from) provision for income taxes
Benefit from (provision for) income taxes
Income Tax Expense (Benefit)
Research and development
Research and Development Expense [Member]
Research and Development Expense
Reductions for tax positions of prior years
Unrecognized Tax Benefits, Decrease Resulting from Prior Period Tax Positions
Summary of Aggregate Payments under Contractually Binding Manufacturing and Service Agreements
Contractual Obligation, Fiscal Year Maturity [Table Text Block]
Leases
Lessee, Leases [Policy Text Block]
Royalty revenue
Non-cash Collaboration Royalty Revenue
Royalty [Member]
Stock-based compensation expense
Share-Based Payment Arrangement, Expense
Disaggregation Of Revenue [Table]
Disaggregation of Revenue [Table]
Option vesting rights, percentage
Share-Based Compensation Arrangement by Share-Based Payment Award, Award Vesting Rights, Percentage
Liabilities for sales of future royalties.
Liabilities for Sales of Future Royalties [Table]
Selling, general and administrative
Collaborative Arrangements Effective Reduction Of Expense In Selling General And Administrative Expenses
Collaborative arrangements effective reduction of expense in selling, general and administrative expenses.
Finite-Lived Intangible Assets, Net, Beginning Balance
Finite-Lived Intangible Assets, Net, Ending Balance
Finite-Lived Intangible Assets, Net
Total
Unrealized gain (loss) on securities available-for-sale
AOCI, Debt Securities, Available-for-sale, Adjustment, after Tax, Total
Unrealized gain on securities available-for-sale
AOCI, Debt Securities, Available-for-Sale, Adjustment, after Tax
Revenue
Revenue from Contract with Customer [Text Block]
Effective income tax rate unrecognized tax benefits
Effective Income Tax Rate Reconciliation, Change in Enacted Tax Rate, Amount
Share-based Compensation Arrangement by Share-based Payment Award, Options, Grants in Period, Net of Forfeitures, Total
Number of Options, Options granted
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Grants in Period, Net of Forfeitures
Impairment of Long-Lived Assets
Impairment or Disposal of Long-Lived Assets, Policy [Policy Text Block]
Other comprehensive income (loss):
Other Comprehensive Income (Loss), Net of Tax [Abstract]
Less: Amount representing interest
Finance Lease, Liability, Undiscounted Excess Amount
Aggregate Intrinsic Value, Outstanding
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Intrinsic Value
Schedule Of Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Table]
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Table]
Debt instrument, face amount
Debt Instrument, Face Amount
Net Loss per Share
Earnings Per Share, Policy [Policy Text Block]
Unrecognized tax benefit
Additions based on tax positions related to current year
Unrecognized Tax Benefits, Increase Resulting from Current Period Tax Positions
Effective Income Tax Rate Reconciliation, Tax Credit, Percent, Total
Federal tax credits
Effective Income Tax Rate Reconciliation, Tax Credit, Percent
Royalty Purchase Agreement
Royalty Purchase Agreement [Member]
Royalty purchase agreement.
Other expense
Other Nonoperating Income (Expense), Total
Other Nonoperating Income (Expense)
Public offering price
Sale of Stock, Price Per Share
Arcturus
Arcturus Research Collaboration And License Agreement [Member]
Arcturus research collaboration and license agreement.
Arcturus Therapeutics Holdings Inc
Operating
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2028
Operating And Finance Lease Liability Payments Due Year Five
Operating and finance lease liability payments due year five.
Operating expenses:
Operating Expenses [Abstract]
Property, Plant and Equipment [Abstract]
Premium from stock purchase agreement
Premium From Stock Purchase Agreement
Premium from stock purchase agreement.
Bayer HealthCare LLC
Bayer Health Care L L C [Member]
Bayer Health Care LLC.
First Anniversary
First Anniversary [Member]
First anniversary.
Entity Voluntary Filers
Entity Voluntary Filers
Subsequent Events [Abstract]
Summary of Cash Equivalents and Marketable Debt Securities Classified as Available For Sale Securities
Debt Securities, Available-for-Sale [Table Text Block]
Foreign
Income (Loss) from Continuing Operations before Income Taxes, Foreign
Commitments and Contingencies Disclosure [Abstract]
Other Assets, Noncurrent, Total
Other assets
Other Assets, Noncurrent
Number of shares purchased on exercise of common stock option
Number Of Shares Purchased On Exercise Of Common Stock Option
Number of shares purchased on exercise of common stock option
Employment Inducement Plan
Employment Inducement Plan [Member]
Employment inducement plan.
Balance of product sales reserve at beginning of year
Balance of product sales reserve at end of year
Sales Reserves
Sales reserves.
Other
Other Accrued Liabilities, Current
Thereafter
Finance Lease, Liability, to be Paid, after Year Five
Equity
Equity [Text Block]
Annual license fee payment
Annual License Fee Payment
Annual license fee payment
Payment From Sale Of Future Royalties.
Payment From Sale Of Future Royalties
Payment from sale of future royalties
2027
Finite-Lived Intangible Asset, Expected Amortization, Year Four
2024
Finite-Lived Intangible Asset, Expected Amortization, Year One
Additional Paid in Capital, Ending Balance
Additional Paid in Capital, Beginning Balance
Additional Paid in Capital, Total
Additional paid-in capital
Additional Paid in Capital
Inventory, Net [Abstract]
Gross Unrealized Gains
Debt Securities, Available-for-Sale, Accumulated Gross Unrealized Gain, before Tax
Extinguishment of Debt [Axis]
Stockholders’ equity:
Equity, Attributable to Parent [Abstract]
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Investment amount
Investment Owned, Fair Value
Investment Owned, at Fair Value, Beginning Balance
Investment Owned, at Fair Value, Ending Balance
Thereafter
Lessee, Operating Lease, Liability, to be Paid, after Year Five
Summary of Components of Lease Expense
Lease, Cost [Table Text Block]
Entity [Domain]
Entity
Common stock, Transfer of value upon option exercise
Transfer Of Fair Value On Exercise Of Options
Transfer of fair value on exercise of options.
Research Collaboration and License Agreement Commercial Milestones
Research Collaboration And License Agreement Commercial Milestones [Member]
Research collaboration and license agreement commercial milestones.
Total estimated fair value of options vested
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested in Period, Fair Value
Earnings Per Share [Abstract]
Lessee, Operating Lease, Liability, to be Paid, Maturity [Table Text Block]
Schedule of Maturities of Lease Liabilities and the Reconciliation of Lease Liabilities
Outstanding cash and cash equivalents acquired
Outstanding Cash and Cash Equivalents Acquired
Outstanding Cash and Cash Equivalents Acquired
Proceeds from sale of future royalties
Proceeds From Sale Of Future Royalties
Proceeds from sale of future royalties.
Deferred Tax Liabilities, Net, Total
Net deferred tax liabilities
Deferred Tax Liabilities, Net
Accounting Policies [Abstract]
Fixed assets and intangibles
Deferred Tax Asset Fixed Assets And Intangibles
Deferred tax asset fixed assets and intangibles.
Manufacturing and Services
Manufacturing And Services [Member]
Manufacturing and services.
Lessee Disclosure [Abstract]
2028
Finite-Lived Intangible Asset, Expected Amortization, Year Five
In-process Research and Development [Member]
IPR&D Assets
In Process Research and Development [Member]
Latin America [Member]
Latin America
Mahzi Therapeutics, Inc.
Mahzi Therapeutics Inc.
Mahzi Therapeutics Inc [Member]
2025
Finance Lease, Liability, to be Paid, Year Two
Share Based Compensation Arrangement By Share Based Payment Award [Line Items]
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items]
Available-for-sale securities remaining contractual maturities
Available For Sale Securities Contractual Maturity
Available for sale securities contractual maturity.
Effective annual interest rate
Debt Instrument, Interest Rate, Effective Percentage
Net loss per share, basic
Earnings Per Share, Basic, Total
Earnings Per Share, Basic
Schedule of Total Accumulated Other Comprehensive Income (Loss)
Schedule of Accumulated Other Comprehensive Income (Loss) [Table Text Block]
Commitments and Contingencies
Commitments and contingencies (Notes 9 and 15)
Business Combinations [Abstract]
Income Statement [Abstract]
Uncertain tax positions
Effective Income Tax Rate Reconciliation Change In Uncertain Tax Positions
Effective income tax rate reconciliation change in uncertain tax positions.
Saint Louis University License Agreement [Member]
Saint Louis University license agreement.
Saint Louis University
Debt Securities, Available-for-sale, Current, Total
Marketable debt securities
Debt Securities, Available-for-Sale, Current
Income Taxes Disclosure [Table]
Income Taxes Disclosure [Table]
Income taxes disclosure.
Commercial Paper
Commercial Paper, Not Included with Cash and Cash Equivalents [Member]
Related Party
Related Party, Type [Axis]
Research Collaboration and License Agreement Development Milestones
Research Collaboration And License Agreement Development Milestones [Member]
Research collaboration and license agreement development milestones.
Percentage of right to receive royalty payments on net sales
Percentage of right to receive royalty payments on net sales.
Percentage of right to receive royalty payments on net sales
Performance Stock Units
Performance Shares [Member]
Royalty Revenue in European Territory
Royalty Revenue in European Territory
European Union And Switzerland And Turkey [Member]
European Union and Switzerland and Turkey.
Summary of Changes in Contract Liabilities
Contract with Customer, Contract Asset, Contract Liability, and Receivable [Table Text Block]
Operating lease expense
Operating Lease, Cost
Unrecognized Tax Benefits, Income Tax Penalties and Interest Expense, Total
Unrecognized tax benefits related to interest or penalties
Unrecognized Tax Benefits, Income Tax Penalties and Interest Expense
Vested and expected to vest December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested and Expected to Vest, Outstanding, Number
Statistical Measurement
Statistical Measurement [Domain]
License and Technology Access Agreement
License And Technology Access Agreement [Member]
License and technology access agreement.
Net cash used in operating activities
Net Cash Provided by (Used in) Operating Activities
Baylor Research Institute License Agreement [Member]
Baylor research institute license agreement.
Baylor Research Institute
2014 Equity Incentive, 2023 Incentive and Employment Inducement Plan
Two Thousand Fourteen And Two Thousand Twenty Three And Employment Inducement Plan [Member]
Two thousand fourteen and two thousand twenty three and employment inducement plan.
Increase (Decrease) in Deferred Liabilities, Total
Deferred tax liabilities
Increase (Decrease) in Deferred Liabilities
Contractual Obligation Maturity Period
Contractual Obligation Maturity Period
Issuance of common stock in connection with underwritten public offering, net of issuance costs
Stock Issued During Period Value Secondary Issues
Stock issued during period value secondary issues.
Schedule of Changes in Fair Value of Equity Investment
Schedule Of Changes In Fair Value Of Equity Investment Table [Text Block]
Schedule of changes in fair value of equity investment.
Aggregate intrinsic value
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Aggregate Intrinsic Value, Vested
Research, clinical study, and manufacturing expenses
Research Clinical Study And Manufacturing Expenses
Research, clinical study, and manufacturing expenses.
Document Period End Date
Document Period End Date
Statistical Measurement
Statistical Measurement [Axis]
Acquisition Costs, Period Cost
Acquisition cost
Accounts Receivable, Allowance for Credit Loss, Ending Balance
Accounts Receivable, Allowance for Credit Loss, Beginning Balance
Allowance for doubtful accounts
Accounts Receivable, Allowance for Credit Loss
Commitments and Contingencies
Commitments and Contingencies Disclosure [Text Block]
Cash paid for measurement of operating lease liabilities
Operating Lease, Payments
Non-cash royalty revenue
Non Cash Royalty [Member]
Non cash royalty.
Net loss per share, diluted
Earnings Per Share, Diluted, Total
Earnings Per Share, Diluted
Product and Service
Product and Service [Domain]
Common stock, Acquisition of investment in securities
Acquisition Of Investment In Common Stock
Acquisition of investment in common stock.
Loss carryforwards
Deferred tax assets net operating loss, federal tax credits
Deferred Tax Assets, Operating Loss Carryforwards, Domestic
Lease, Cost [Abstract]
Cost of sales
Cost of Sales [Member]
Deferred Tax Assets, Operating Loss Carryforwards, Total
Deferred tax assets net operating loss carryforwards
Loss carryforwards
Deferred Tax Assets, Operating Loss Carryforwards
Balance at beginning of year
Balance at end of year
Unrecognized Tax Benefits
2026
Contractual Obligation, to be Paid, Year Three
Royalty agreement termination threshold amount
Royalty Agreement Termination Threshold Amount
Royalty agreement termination threshold amount.
Purchase price of shares purchased on exercise of common stock option
Purchase Price Of Shares Purchased On Exercise Of Common Stock Option
Purchase price of shares purchased on exercise of common stock option.
Collaboration Revenue
Collaboration [Member]
Collaboration.
Total revenues
Revenue from Contract with Customer [Abstract]
Fair Value Hierarchy and NAV
Fair Value Hierarchy and NAV [Domain]
Vested and exercisable December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Exercisable, Number
2028
Finance Lease, Liability, to be Paid, Year Five
Deferred compensation liabilities
Deferred Compensation Liabilities [Member]
Deferred compensation liabilities.
Related Party Transaction [Domain]
Number of reportable segments
Number of Reportable Segments
Other Commitments
Other Commitments [Domain]
Inventory
Inventory, Policy [Policy Text Block]
Cumulative foreign currency translation adjustment
Accumulated Other Comprehensive Income (Loss), Foreign Currency Translation Adjustment, Net of Tax
Accumulated Other Comprehensive Income (Loss), Foreign Currency Translation Adjustment, Net of Tax, Ending Balance
Accumulated Other Comprehensive Income (Loss), Foreign Currency Translation Adjustment, Net of Tax, Beginning Balance
Components of Deferred Tax Assets and Liabilities [Abstract]
2011 Equity Incentive, 2014 Incentive, 2023 Incentive and Employment Inducement Plan
Two Thousand Eleven And Two Thousand Fourteen And Two Thousand Twenty Three And Employment Inducement Plan [Member]
Two thousand eleven and two thousand fourteen and two thousand twenty three and employment inducement plan.
Mepsevii
Mepsevii [Member]
Mepsevii.
Finite-Lived Intangible Assets, Major Class Name
Finite-Lived Intangible Assets, Major Class Name [Domain]
Number of Options, Options exercised
Number of option exercised
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Exercises in Period
Basis of Consolidation
Consolidation, Policy [Policy Text Block]
Income Taxes Disclosure [Line Items]
Income Taxes Disclosure [Line Items]
Income taxes disclosure.
LIABILITIES AND STOCKHOLDERS' EQUITY
Liabilities and Equity [Abstract]
Operating Lease, Liability, Statement of Financial Position [Extensible List]
Operating Lease, Liability, Current, Statement of Financial Position [Extensible Enumeration]
Research and development
Collaborative Arrangements Effective Reduction Of Expense In Research And Development Expenses
Collaborative arrangements effective reduction of expense in research and development expenses.
Profit Share Territory
United States And Canada [Member]
United States and Canada.
Option to sell common stock aggregate offering price
Option To Sell Common Stock Aggregate Offering Price
Option to sell common stock aggregate offering price.
Milestone payments received
Milestone Payments Received
Milestone payments received.
Entity Address, Postal Zip Code
Entity Address, Postal Zip Code
Entity Interactive Data Current
Entity Interactive Data Current
Proceeds from the issuance of common stock in connection with at-the-market offering, net of issuances costs
Proceeds from the Issuance of Common Stock in Connection with at-the-Market Offering, Net of Issuances Costs
Proceeds from the issuance of common stock in connection with at-the-market offering, net of issuances costs.
2026
Finite-Lived Intangible Asset, Expected Amortization, Year Three
Equity [Abstract]
Less: Amount representing interest
Lessee, Operating Lease, Liability, Undiscounted Excess Amount
Related party transaction contribution period.
Related Party Transaction Contribution Period
Contribution period
Schedule of Indefinite-Lived Intangible Assets [Table]
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Grants in Period
PSOs granted
Number of shares, granted
Entity Well-known Seasoned Issuer
Entity Well-known Seasoned Issuer
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Abstract]
Weighted Average Exercise Price, Options exercised
Share-Based Compensation Arrangements by Share-Based Payment Award, Options, Exercises in Period, Weighted Average Exercise Price
Entity Incorporation, State or Country Code
Entity Incorporation, State or Country Code
Liabilities for sales of future royalties.
Liabilities for Sales of Future Royalties [Text Block]
Liabilities for Sales of Future Royalties
Two Thousand Twenty Three Incentive Plan [Member]
2023 Incentive Plan
Two thousand twenty three incentive plan.
Equity Components
Equity Components [Axis]
Accounts receivable
Increase (Decrease) in Accounts Receivable
Investments, Debt and Equity Securities [Abstract]
Payments to Acquire Businesses, Gross
Payments to Acquire Businesses, Gross
Shares available for grant, ending date
Share Based Compensation Arrangement By Share Based Payment Award Shares Available For Grant Ending Date
Share based compensation arrangement by share based payment award shares available for grant ending date.
Regeneron pharmaceuticals Inc.
Regeneron Pharmaceuticals Inc [Member]
Regeneron Pharmaceuticals Inc
Furniture and Office Equipment
Office Equipment [Member]
GeneTx [Member]
GeneTx [Member]
GeneTx [Member]
Weighted-Average Grant Date Fair Value, vested
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Vested in Period, Weighted Average Grant Date Fair Value
Local Phone Number
Local Phone Number
Indefinite-Lived Intangible Assets [Member]
Indefinite-lived Intangible Assets
Property Plant And Equipment [Line Items]
Property, Plant and Equipment [Line Items]
Sale of Stock
Sale of Stock [Axis]
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Exercisable, Weighted Average Exercise Price
Weighted- Average Exercise Price,Weighted Average Exercise Price
Deferred compensation
Deferred Compensation
Deferred compensation.
Debt conversion, converted instrument, shares issued
Debt Conversion, Converted Instrument, Shares Issued
2026
Operating And Finance Lease Liability Payments Due Year Three
Operating and finance lease liability payments due year three.
Convertible Promissory Note
Convertible Promissory Note [Member]
Convertible promissory note.
Marketable Debt Securities
Available For Sale Securities Debt Securities [Policy Text Block]
Disclosure of accounting policy for available for sale securities debt securities.
Equity Securities, FV-NI, Gain (Loss), Total
Change in fair value of equity investments
Change in fair value of equity investments
Equity Securities, FV-NI, Gain (Loss)
Schedule of finite and indefinite lived intangible asset.
Schedule of Finite and Indefinite Lived Intangible Assets [Line Items]
Goodwill, Ending Balance
Goodwill, Beginning Balance
Goodwill, Total
Goodwill
Goodwill
Restricted cash included in other current assets
Restricted Cash, Current
Statement of Cash Flows [Abstract]
Comprehensive Loss
Comprehensive Income, Policy [Policy Text Block]
Income Tax Authority
Income Tax Authority [Domain]
Legally-binding minimum lease payments.
Legally binding Minimum Lease Payments
Legally binding minimum lease payments
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Total accumulated other comprehensive income (loss)
Accumulated other comprehensive income (loss)
Accumulated Other Comprehensive Income (Loss), Net of Tax
Property, Plant, and Equipment
Property, Plant and Equipment, Policy [Policy Text Block]
Document Annual Report
Document Annual Report
License and Research Agreements
Collaborative Arrangement And Noncollaborative Arrangement Transactions Disclosure [Text Block]
Collaborative arrangement and noncollaborative arrangement transactions disclosure.
Common stock. per share
Common stock, par value
Common Stock, Par or Stated Value Per Share
Effect of Exchange Rate on Cash, Cash Equivalents, Restricted Cash and Restricted Cash Equivalents, Including Disposal Group and Discontinued Operations, Total
Effect of exchange rate changes on cash
Effect of Exchange Rate on Cash, Cash Equivalents, Restricted Cash, and Restricted Cash Equivalents, Including Disposal Group and Discontinued Operations
Numerator:
Net Income (Loss) Available to Common Stockholders, Basic [Abstract]
Weighted-average period to recognize cost
Share-Based Payment Arrangement, Nonvested Award, Cost Not yet Recognized, Period for Recognition
Long-Lived Tangible Asset
Long-Lived Tangible Asset [Axis]
2023 Plan and Inducement Plan
Two Thousand And Twenty Three Incentive Plan And Employment Inducement Plan [Member]
Two thousand and twenty three incentive plan and employment inducement plan.
Weighted-Average Remaining Contractual Term,Vested and expected to vest December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested and Expected to Vest, Outstanding, Weighted Average Remaining Contractual Term
Share of common stock authorized for future issuance
Share-Based Compensation Arrangement by Share-Based Payment Award, Number of Shares Authorized
Effective Income Tax Rate Reconciliation, Percent [Abstract]
Schedule Of Share Based Compensation Arrangements By Share Based Payment Award [Table]
Schedule of Share-Based Compensation Arrangements by Share-Based Payment Award [Table]
Maximum
Maximum [Member]
Issuance of common stock and pre-funded warrants in connection with underwritten public offering, net of issuance costs,shares
Pre-funded Warrants And Stock Issued During Period Shares At Underwritten Public Offering
Pre-funded warrants and stock issued during period shares at underwritten public offering.
Development Costs, Period Cost
Development costs
2024
Contractual Obligation, to be Paid, Year One
Related Party Transaction [Axis]
Research and Development Equipment
Research And Development Equipment [Member]
Research and development equipment.
OMERS [Member]
OMERS [Member]
OMERS
Debt Securities in Government Sponsored Entities
Debt Securities In Government Sponsored Entities [Member]
Debt securities in government sponsored entities.
Unrecognized Tax Benefits
Schedule of Unrecognized Tax Benefits Roll Forward [Table Text Block]
Schedule of Sales-Related Accruals and Allowances
Schedule Of Sales Related Accruals And Allowances Table [Text Block]
Schedule of sales-related ccruals and allowances.
Fair value of option to purchase common stock, Change in fair value
Change in Fair Value of Common Stock
Change in fair value of common stock.
Level 3
Fair Value, Inputs, Level 3 [Member]
Stock-based compensation
APIC, Share-based Payment Arrangement, Increase for Cost Recognition, Total
APIC, Share-Based Payment Arrangement, Increase for Cost Recognition
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Table]
Fair Value, Recurring and Nonrecurring [Table]
Total operating expenses
Cost of sales and operating expenses.
Cost Of Sales And Operating Expenses
Short-term Debt, Type
Short-Term Debt, Type [Domain]
Investment in solid common stock member.
Investment in Solid Common Stock [Member]
Investments in Solid Common Stock
Allocated fair value of warrant
Fair Value Adjustment of Warrants
Net increase (decrease) in cash, cash equivalents, and restricted cash
Cash, Cash Equivalents, Restricted Cash, and Restricted Cash Equivalents, Period Increase (Decrease), Including Exchange Rate Effect
Payables and Accruals [Abstract]
Other
Payments for (Proceeds from) Other Investing Activities
Proceeds from issuance of common stock in connection with license agreement
Proceeds from the issuance of common stock in connection with the license agreement, net
Proceeds From Issuance Of Common Stock In Connection With License Agreement
Proceeds from issuance of common stock in connection with license agreement.
Intangible Assets, Gross (Excluding Goodwill), Total
Intangible Assets, Gross (Excluding Goodwill)
Intangible assets - Gross carrying amount
upfront payment.
upfront payment
upfront payment
Number of Options, Options cancelled
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Forfeitures in Period
Program Agreement And Unitholder Option Agreement Member
Program Agreement And Unitholder Option Agreement [Member]
Program agreement and unitholder option agreement.
Accrued Liabilities, Total
License agreement accrued liabilities
Accrued Liabilities
Document Financial Statement Error Correction [Flag]
Non-cash collaboration royalty revenue
Non-cash collaboration royalty revenue
Non Cash Collaboration Royalty Revenue
Non cash collaboration royalty revenue.
Outstanding Shares of Common Stock Equivalents Excluded from Computation of Diluted Net Loss per Share
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share [Table Text Block]
Summary of Inventory
Schedule of Inventory, Current [Table Text Block]
City Area Code
City Area Code
Research and Development Arrangement, Contract to Perform for Others, Type
Research and Development Arrangement, Contract to Perform for Others, Type [Axis]
Deferred income tax expense
State
Deferred income tax benefit
Deferred State and Local Income Tax Expense (Benefit)
Total inventory
Inventory
Inventory, Net
Operating activities:
Net Cash Provided by (Used in) Operating Activities [Abstract]
2014 Employee Stock Purchase Plan
Two Thousand Fourteen Employee Stock Purchase Plan [Member]
Two thousand fourteen employee stock purchase plan.
Aggregate Intrinsic Value, Outstanding
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Aggregate Intrinsic Value, Outstanding
Share of Collaboration and Royalty Revenue Related to Crysvita
Schedule Of Collaboration And Royalty Revenue Table [Text Block]
Schedule of collaboration and royalty revenue.
Business Acquisition Line Items
Business Acquisition [Line Items]
Basis Difference in Equity Investments
Equity Method Investments [Member]
Customer [Domain]
Accumulated Depreciation, Depletion and Amortization, Property, Plant, and Equipment, Ending Balance
Accumulated Depreciation, Depletion and Amortization, Property, Plant, and Equipment, Beginning Balance
Accumulated Depreciation, Depletion and Amortization, Property, Plant, and Equipment, Total
Less accumulated depreciation
Accumulated Depreciation, Depletion and Amortization, Property, Plant, and Equipment
JAPAN
Japan
Selling, general and administrative
Selling, General and Administrative Expenses [Member]
Options to Purchase Common Stock, RSUs, and PSUs
Options To Purchase Common Stock Restricted Stock Units And Performance Stock Units [Member]
Options to purchase common stock, restricted stock units and performance stock units.
Shares used in computing net loss per share, Basic
Weighted Average Number of Shares Outstanding, Basic, Total
Weighted Average Number of Shares Outstanding, Basic
Weighted-average shares used to computing net loss per share, basic
REGENXBIO, Inc.
R E G E N X B I O Incorporated [Member]
REGENXBIO, incorporated.
Research and Development Arrangement, Contract to Perform for Others, Type
Research and Development Arrangement, Contract to Perform for Others, Type [Domain]
Work-in-process
Inventory, Work in Process, Gross
Employee Stock Purchase Plan
Employee Stock [Member]
Financing activities:
Net Cash Provided by (Used in) Financing Activities [Abstract]
Research and Development
Research and Development Expense, Policy [Policy Text Block]
Foreign Income Tax Expense (Benefit), Continuing Operations, Total
International
Foreign Income Tax Expense (Benefit), Continuing Operations
Development milestone payments
Development Milestone Payments
Development milestone payments.
Contract With Customer Assets Liability Current
Contract with customer assets (liability) current.
Balance of contract liabilities at beginning of period
Balance of contract liabilities at end of period, net
Summary of Activity Under Stock Units from 2014 Plan
Share-Based Payment Arrangement, Restricted Stock Unit, Activity [Table Text Block]
Stock sold in at-the-market offering
Issuance of common stock in connection with at-the-market offering, net of issuance costs, shares
Stock Issued During Period Shares At Market Offering
Stock issued during period shares at market offering.
Statement [Table]
Statement [Table]
Summary of Activity Under Stock Option Plans Including 2011 Plan, 2014 Plan and 2023 Plan
Share-Based Payment Arrangement, Option, Activity [Table Text Block]
Schedule of Related Party Transactions, by Related Party [Table]
International
Foreign Jurisdiction [Member]
Foreign Jurisdiction.
Document Fiscal Period Focus
Document Fiscal Period Focus
Total future lease payments
Lessee, Operating Lease, Liability, to be Paid
Shares purchased by underwriters
Stock Purchased By Underwriters Pursuant To Option Granted
Stock purchased by underwriters pursuant to option granted.
Related Party Transactions Disclosure [Text Block]
Related Party Transaction
Tax Credit Carryforward [Line Items]
Tax Credit Carryforward [Line Items]
2025
Lessee, Operating Lease, Liability, to be Paid, Year Two
Statement [Line Items]
Statement [Line Items]
Dia Carta Ltd
Dia Carta Ltd [Member]
DiaCarta Ltd.
Schedule of finite and indefinite lived intangible assets.
Schedule of Finite and Indefinite Lived Intangible Assets [Table]
Common stock equivalents excluded from computation of diluted net loss per share
Antidilutive Securities Excluded from Computation of Earnings Per Share, Amount
Organization And Nature Of Business [Table]
Organization And Nature Of Business [Table]
Organization and nature of business.
Total
Lease, Cost
Solid Biosciences, Inc.
Solid Biosciences Inc [Member]
Solid Biosciences, Inc.
Schedule Of Collaboration Cost Sharing and Payments
Schedule Of Collaboration Cost Sharing and Payments [Table Text Block]
Schedule of collaboration cost sharing and payments.
Asset Class
Asset Class [Domain]
Schedule of Tax Effect of Temporary Differences to Significant Portions of Deferred Tax Assets
Schedule of Deferred Tax Assets and Liabilities [Table Text Block]
Accounts Receivable, after Allowance for Credit Loss, Current, Total
Accounts receivable, net
Accounts Receivable, after Allowance for Credit Loss, Current
Future contingent milestone payments.
Future Contigent Milestone Payments
Future contingent milestone payments
Customer [Axis]
Common Stock
Common Stock [Member]
Thereafter
Operating And Finance Lease Liability Due After Year Five
Operating and finance lease liability due after year five.
Cash and Cash Equivalents, at Carrying Value, Ending Balance
Cash and Cash Equivalents, at Carrying Value, Beginning Balance
Cash and Cash Equivalents, at Carrying Value, Total
Cash and cash equivalents
Cash and Cash Equivalents, at Carrying Value
Proceeds from the issuance of common stock and pre-funded warrants in connection with underwritten public offerings, net of issuance costs
Proceeds from the Issuance Of Common Stock and Pre-funded Warrants in Connection with Underwritten Public Offerings, Net Of Issuance Costs
Proceeds from the issuance of common stock and pre-funded warrants in connection with underwritten public offerings, net of issuance costs.
Summary of Stock-Based Compensation Expense
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Table Text Block]
Option to sell common stock for cash
Option To Sell Common Stock For Cash
Option to sell common stock for cash.
Other assets
Other Assets, Current
Corporate Bonds
Corporate Bond Securities [Member]
Amortization of premium (discount) on marketable debt securities, net
Accretion (Amortization) of Discounts and Premiums, Investments
Manufacturing Equipment
Manufacturing Facility [Member]
Building
Building [Member]
Entity Common Stock, Shares Outstanding
Entity Common Stock, Shares Outstanding
Proceeds from sale of marketable debt securities
Proceeds from Sale of Debt Securities, Available-for-Sale
Inventory, Finished Goods, Gross, Total
Finished goods
Inventory, Finished Goods, Gross
Antidilutive Securities, Name
Antidilutive Securities, Name [Domain]
Domestic
Income (Loss) from Continuing Operations before Income Taxes, Domestic
Stock Purchase Agreement
Stock Purchase Agreement [Member]
Stock purchase agreement.
Liabilities for sales of future royalties
Liabilities for Sales of Future Royalties Current
Liabilities for sales of future royalties current
Financing:
Financing Lease [Abstract]
Financing lease.
Treasury stock, at cost, 9,559 in 2023 and nil in 2022
Treasury Stock, Value
Treasury Stock, Value, Total
Cover
Cover [Abstract]
Selling, General and Administrative Expense, Total
Selling, general and administrative
Selling, General and Administrative Expense
Net carrying value of investments
Net Carrying Value of Investments
Net carrying value of investments.
Vesting
Vesting [Axis]
Stockholders equity note number of common shares held after split.
Stockholders Equity Note Number of Common Shares Held After Split
Number of common shares held after split
Document Fiscal Year Focus
Document Fiscal Year Focus
Accumulated Other Comprehensive Income (Loss)
Comprehensive Income (Loss) Note [Text Block]
Future Minimum Lease Payments - Operating and Financing Leases
Operating And Financing Lease [Abstract]
Operating and financing lease.
Kyowa Kirin Collaboration
Kyowa Kirin Collaboration [Member]
Kyowa Kirin Collaboration.
State and Local Income Tax Expense (Benefit), Continuing Operations, Total
State
State and Local Income Tax Expense (Benefit), Continuing Operations
Sale of Stock
Sale of Stock [Domain]
Accumulated Other Comprehensive Income (Loss), Net of Tax [Abstract]
Preferred Stock
Preferred Stock [Member]
Stock-Based Compensation
Share-Based Payment Arrangement [Policy Text Block]
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Grants in Period, Weighted Average Grant Date Fair Value
Weighted Average Exercise Price,PSOs granted
Weighted-Average Grant Date Fair Value, granted
Proceeds, Issuance of Shares, Share-based Payment Arrangement, Including Option Exercised, Total
Proceeds from the issuance of common stock from exercise of warrants and equity plan awards, net
Proceeds, Issuance of Shares, Share-Based Payment Arrangement, Including Option Exercised
Regulatory milestone payments
Regulatory Milestone Payments
Regulatory milestone payments.
Non-cash interest expense on liability related to the sale of future royalties capitalized into ending property, plant and equipment
Non-cash interest expense on liability related to the sale of future royalties capitalized into ending property, plant and equipment
Non-cash interest expense on liabilities for sales of future royalties capitalized during the year into ending property, plant and equipment
Security Exchange Name
Security Exchange Name
Award expiration period
Share-Based Compensation Arrangement by Share-Based Payment Award, Expiration Period
Summary of Company's Intangible Assets
Schedule of intangible assets.
Schedule of Intangible Assets [Table Text Block]
Federal
Domestic Tax Authority [Member]
Contract liabilities
Contract Liabilities Current
Contract liabilities current.
Preferred stock, shares authorized
Preferred Stock, Shares Authorized
Schedule Of Finite Lived Intangible Assets [Table]
Schedule of Finite-Lived Intangible Assets [Table]
Concentration of Credit Risk, Credit Losses, and Other Risks and Uncertainties
Concentration Of Credit Risk Credit Losses And Other Risks And Uncertainties Policy [Text Block]
Concentration of credit risk credit losses and other risks and uncertainties.
Property, Plant and Equipment, Gross, Ending Balance
Property, Plant and Equipment, Gross, Beginning Balance
Property, Plant and Equipment, Gross, Total
Property, Plant and Equipment, Gross
Property, plant and equipment, gross
Fit - out costs.
Fit out Costs
Fit out costs
Regulatory milestone payments achieved
RegulatoryMilestonePaymentsAchieved
Regulatory milestone payments achieved.
Liability for sale of future royalties.
Liability For Sale Of Future Royalties
Liability for sale of future royalties at beginning of year
Liability for sale of future royalties at end of year
Weighted Average Exercise Price, Options cancelled
Share-Based Compensation Arrangements by Share-Based Payment Award, Options, Forfeitures in Period, Weighted Average Exercise Price
Cost of goods sold
Cost of sales
Cost Of Sales
Cost of sales.
Business Acquisition, Acquiree
Business Acquisition, Acquiree [Domain]
Legal Entity [Axis]
Legal Entity
Deferred compensation liabilities
Deferred Compensation Liability, Current and Noncurrent
Deferred Compensation Liability, Current and Noncurrent, Total
Financial Instrument [Axis]
Number of common stock purchased
Number Of Common Stock Purchased
Number of common stock purchased.
Denominator:
Weighted Average Number of Shares Outstanding, Diluted [Abstract]
Liability for sale of future royalties.
Liability For Sale Of Future Royalties [Line Items]
Liability For Sale Of Future Royalties [Line Items]
2024
Finance Lease, Liability, to be Paid, Year One
Common stock, Purchase price of option exercise
Stock Issued During Period, Value, Stock Options Exercised
Entity Emerging Growth Company
Entity Emerging Growth Company
Total intrinsic value of options exercised
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Exercises in Period, Intrinsic Value
Amendment
Amendment Flag
Amendment Flag
Summary of Accrued Liabilities
Schedule of Accrued Liabilities [Table Text Block]
Payments to Acquire Intangible Assets
Payment for intangible assets
Payment for intangible asset
Gross Unrealized Losses
Gross Unrealized Losses
Debt Securities, Available-for-Sale, Accumulated Gross Unrealized Loss, before Tax
Abeona [Member]
Abeona [Member]
Abeona
Operating loss carryforwards expiration year
Operating Loss Carryforwards Expiration Year
Operating loss carryforwards expiration year.
Eligible future milestone payments
Eligible Future Milestone Payments
Eligible future milestone payments.
Amortization
Finance Lease, Right-of-Use Asset, Amortization
Schedule of Finite-Lived Intangible Assets, Future Amortization Expense [Table Text Block]
Schedule of Expected Amortization of the Intangible Assets
Deferred tax liabilities:
Deferred Tax Liabilities, Gross [Abstract]
Liability related to the sale of future royalties at beginning of year
Liability related to the sale of future royalties at end of year
Liability Related To Sale Of Future Royalties
Liability related to sale of future royalties.
Liabilities for sales of future royalties
Computer Equipment and Software
Computer Equipment And Software [Member]
Computer equipment and software.
Purchase of marketable debt securities
Payments to Acquire Debt Securities, Available-for-Sale
Stock options
Deferred Tax Assets Stock Option
Deferred tax assets stock option.
Deductions
Contract With Customer Liability Deductions During Period
Contract with customer liability deductions during period.
Finite-Lived Intangible Asset, Expected Amortization, after Year Five
Thereafter
Proceeds from sale of equity investments
Proceeds From Sale Of Equity Investment
Proceeds from sale of equity investment.
Entity File Number
Entity File Number
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Shares Vesting Monthly After First Anniversary
Shares Vesting Monthly After First Anniversary [Member]
Shares vesting monthly after first anniversary.
2025
Contractual Obligation, to be Paid, Year Two
Gross deferred tax assets
Deferred Tax Assets, Gross
Financing lease weighted average discount rate
Finance Lease, Weighted Average Discount Rate, Percent
Aggregate Intrinsic Value, Vested and exercisable - December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Vested and Expected to Vest, Exercisable, Aggregate Intrinsic Value
Unrealized gain (loss) on available-for-sale securities
OCI, Debt Securities, Available-for-Sale, Unrealized Holding Gain (Loss), before Adjustment, after Tax
Stock-based compensation
Allocated Share Based Compensation Expense Net Of Capitalized Inventory Cost
Allocated share based compensation expense net of capitalized inventory cost.
Share-Based Payment Arrangement [Abstract]
Additional option to purchase common stock value per share
Option To Purchase Additional Common Stock Value Per Share
Option to purchase additional common stock value per share.
Regulatory approval milestones
Regulatory Approval Milestones
Provisions
Sales Reserves Provisions
Sales reserves, provisions.
Net proceeds from at-the-market offering
Proceeds from the issuance of common stock in connection with at-the-market offering, net
Proceeds From Issuance Of Common Stock At Market Offering
Proceeds from issuance of common stock at market offering.
Annual fees payable
Annual Fees Payable
Annual fees payable.
Accounts Payable, Current, Total
Accounts payable
Accounts Payable, Current
Weighted- Average Exercise Price,Outstanding December 31, 2023
Outstanding, Weighted-Average Grant Date Fair Value, Unvested, Beginning Balance
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Nonvested, Weighted Average Grant Date Fair Value
Acquired in-process research and development
Research and Development in Process 1
Research and development in process.
Deferred Tax Assets, Valuation Allowance, Total
Valuation allowance
Deferred Tax Assets, Valuation Allowance
License agreement other receivables
Other Receivables
Auditor Location
Expected term in years
Share-Based Compensation Arrangement by Share-Based Payment Award, Fair Value Assumptions, Expected Term
Marketable debt securities
Debt Securities, Available-for-Sale, Noncurrent
Preferred stock, par value of $0.001 per share - 25,000,000 shares authorized; nil outstanding in 2023 and in 2022
Preferred Stock, Value, Issued, Ending Balance
Preferred Stock, Value, Issued, Beginning Balance
Preferred Stock, Value, Issued, Total
Preferred Stock, Value, Issued
Entity Small Business
Entity Small Business
Entity Shell Company
Entity Shell Company
Underwritten Public Offering Shares
Sale of Stock, Number of Shares Issued in Transaction
Investment Type
Investment Type [Axis]
Common stock warrants, exercise price
Class of Warrant or Right, Exercise Price of Warrants or Rights
Issuance of common stock under equity plan awards, net of tax, shares
Issuance Of Common Stock Under Equity Plan Awards Net Of Tax Shares
Issuance of common stock under equity plan awards, net of tax, shares.
Class of Warrant or Right
Class of Warrant or Right [Domain]
Profit loss sharing percentage on net sales
Profit Loss Sharing Percentage On Net Sales
Profit loss sharing percentage on net sales.
Net Loss per Share
Earnings Per Share [Text Block]
Revenues:
Revenues [Abstract]
Organization And Nature Of Business [Line Items]
Organization And Nature Of Business [Line Items]
Organization and nature of business.
Entity Address, Address Line One
Entity Address, Address Line One
Contractual Obligation, Total
Contractual Obligation
Total
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items]
Share-Based Payment Arrangement, Expensed and Capitalized, Amount [Line Items]
Antidilutive Securities
Antidilutive Securities [Axis]
Collaboration and License Agreement
Collaboration And License Agreement [Member]
Collaboration and license agreement.
Balance Sheet Components
Supplemental Balance Sheet Disclosures [Text Block]
Royalty revenue
Royalty revenue
Royalty Revenue
Royalty revenue.
Performance stock options.
Performance Stock Options [Member]
Less: Amount representing interest
Operating And Finance Lease Liability Undiscounted Excess Amount
Operating and finance lease liability undiscounted excess amount.
Payments
Sales Reserves Payments
Sales reserves payments.
Income Statement Location
Income Statement Location [Axis]
Number of common stock held
Number of common stock held.
Number Of Common Stock Held
Variable lease expense
Variable Lease, Cost
Percentage of royalty on net sales receives
Royalty Percentage On Net Sales
Royalty percentage on net sales.
Issuance of common stock in connection with license agreement, net of issuance costs
Issuance Of Common Stock Value In Connection With License Agreement
Issuance of common stock value in connection with license agreement.
Schedule of Cost Sharing Payments
Schedule Of Cost Sharing Payments Table [Text Block]
Schedule of cost sharing payments.
Expected dividend rate
Share-Based Compensation Arrangement by Share-Based Payment Award, Fair Value Assumptions, Expected Dividend Rate
2028
Lessee, Operating Lease, Liability, to be Paid, Year Five
Deferred compensation obligation
Deferred Employee Stock Ownership Plan, Issuance of Shares or Sale of Treasury Shares
Level 2
Fair Value, Inputs, Level 2 [Member]
Product Revenue
Product sales
Product [Member]
Common stock shares issued
Share-Based Compensation Arrangement by Share-Based Payment Award, Shares Issued in Period
Collaboration Agreement
Collaboration Agreement [Member]
Collaboration agreement.
Pre-funded warrant price
Share Price
Schedule Of Collaborative Arrangements And Noncollaborative Arrangement Transactions [Table]
Collaborative Arrangement and Arrangement Other than Collaborative [Table]
Depreciation, Depletion and Amortization, Total
Depreciation and amortization
Depreciation, Depletion and Amortization
Product and Service
Product and Service [Axis]
Title of 12(b) Security
Title of 12(b) Security
Finite-Lived Intangible Assets, Accumulated Amortization
Intangible assets - Accumulated amortization
Intangible assets - Accumulated Amortization
Underwritten Public Offering
Underwritten Public Offering [Member]
Underwritten public offering.
Dimension
Dimension
Dimension Therapeutics Inc [Member]
Dimension Therapeutics Inc.
Evkeeza
Evkeeza [Member]
Evkeeza.
Non-cash interest expense
Non Cash Interest Expense On Liability Related To Sale Of Future Royalties
Non cash interest expense on liability related to sale of future royalties.
Non-cash interest expense on liabilities for sales of future royalties
Non-cash interest expense on liabilities for sales of future royalties
Current provision for income taxes:
Current Federal, State and Local, Tax Expense (Benefit) [Abstract]
Fair Value, Assets Measured on Recurring Basis [Table Text Block]
Summary of Financial Assets Measured on Recurring Basis
Investments
Investments [Domain]
Present value of future lease payments
Operating And Finance Lease Liability
Operating and finance lease liability.
Accrued collaboration payables
AccruedCollaborationPayables
Accrued collaboration payables.
Effective Income Tax Rate Reconciliation, Nondeductible Expense, Percent, Total
Nondeductible permanent items
Effective Income Tax Rate Reconciliation, Nondeductible Expense, Percent
Cost Sharing Payments [Abstract]
Cost sharing payments abstract.
Treasury Stock
Treasury Stock, Common [Member]
Investing activities:
Net Cash Provided by (Used in) Investing Activities [Abstract]
Upfront payments of collaboration
Upfront Payments of Collaboration
Upfront payments of collaboration.
State
State and Local Jurisdiction [Member]
Other costs
Other Cost and Expense, Operating
Deferred Compensation Plan
Postemployment Benefit Plans, Policy [Policy Text Block]
Long-Lived Tangible Asset
Long-Lived Tangible Asset [Domain]
Defined Contribution Pension And Other Post Retirement Plan Disclosure [Abstract]
Defined Contribution Pension And Other Post Retirement Plan Disclosure [Abstract]
Tiered double-digit revenue share percentage entitled to receive
Revenue Share Percentage On Commercialized Products Range Mid To High
Revenue share percentage on commercialized products range mid to high.
Orphan Drug Credits [Member]
Orphan Drug Credits [Member]
Orphan drug credits.
Fair value of option to purchase of common stock, Ending balance
Fair value of option to purchase common stock, Beginning balance
Option to Purchase Common Stock Value
Option to purchase common stock value.
Cash, Cash Equivalents, and Restricted Cash
Cash and Cash Equivalents, Restricted Cash and Cash Equivalents, Policy [Policy Text Block]
Increase (Decrease) in Inventories, Total
Inventory
Increase (Decrease) in Inventories
Deferred Revenue balance
Contract with Customer, Liability, Total
Contract with Customer, Liability
Tax Credit Carryforward [Table]
Tax Credit Carryforward [Table]
Issuance of common stock in connection with underwritten public offering, net of issuance costs, shares
Stock Issued During Period Shares Secondary Issues
Stock issued during period shares secondary issues.
Fair value of option to purchase of common stock, Transfer of value upon option exercise
Transfer of Fair Value on Exercise of Options Common Stock
Transfer of fair value on exercise of options common stock.
Weighted Average Remaining Contractual Term (Years), Vested and exercisable - December 31, 2023
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Exercisable, Weighted Average Remaining Contractual Term
Ending balance, shares
Common stock, shares outstanding
Beginning balance, shares
Common Stock, Shares, Outstanding
Risk-free interest rate
Share-Based Compensation Arrangement by Share-Based Payment Award, Fair Value Assumptions, Risk Free Interest Rate
Stock-Based Awards
Share-Based Payment Arrangement [Text Block]
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Forfeitures, Weighted Average Grant Date Fair Value
Weighted Average Exercise Price ,PSOs cancelled
Weighted-Average Grant Date Fair Value, cancelled
Proceeds from Warrant Exercises
Indefinite-Lived Intangible Assets [Line Items]
Tax Credit Carryforward
Tax Credit Carryforward [Axis]
Research Grant Agreement [Abstract]
Research grant agreement.
Income Statement Location
Income Statement Location [Domain]
Weighted Average Exercise Price, Options granted
Share-Based Compensation Arrangements by Share-Based Payment Award, Options, Grants in Period, Weighted Average Exercise Price
License Agreement
License Agreement Terms [Member]
Issuance of common stock and pre-funded warrants in connection with underwritten public offering, net of issuance costs
Pre-funded Warrants And Stock Issued During Period Value At Underwritten Public Offering
Pre-funded warrants and stock issued during period value at underwritten public offering.
Stockholders Equity Line Items
Stockholders Equity [Line Items]
Stockholders equity line items.
Collaboration and Royalty
Collaboration And Royalty [Member]
Collaboration and royalty.
Document Type
Document Type
Intangible Assets, Net (Excluding Goodwill) [Abstract]
Stock-based compensation capitalized to inventory
Employee Service Share Based Compensation Allocation Of Recognized Period Costs Capitalized Amount Including Amount Expensed To Cost Of Goods Sold
Employee service share based compensation allocation of recognized period costs capitalized amount including amount expensed to cost of goods sold.
Additions
Contract With Customer Liability Additions During Period
Contract with customer liability additions during period.
Net cash provided by (used in) investing activities
Net Cash Provided by (Used in) Investing Activities
Counterparty Name
Counterparty Name [Domain]
Rare Disease Education And Awareness [Member]
Rare Disease Education And Awareness [Member]
Rare Disease Education And Awareness
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Forfeited in Period
PSOs cancelled
Number of shares, cancelled
Net cash provided by financing activities
Net Cash Provided by (Used in) Financing Activities
Entity Filer Category
Entity Filer Category
Tax credit carryforwards
Deferred Tax Assets, Tax Credit Carryforwards, Research
Tax credit carryforwards expiration year
Tax Credit Carry Forward Expiration Year
Tax credit carry forward expiration year.
Supplemental disclosures of non-cash investing and financing information:
Cash Flow, Noncash Investing and Financing Activities Disclosure [Abstract]
Foreign currency translation adjustments
Other Comprehensive Income (Loss), Foreign Currency Transaction and Translation Gain (Loss) Arising During Period, Net of Tax
Balance Sheet Location
Balance Sheet Location [Domain]
Components of Company's Loss Before Income Taxes
Schedule of Income before Income Tax, Domestic and Foreign [Table Text Block]
Sublicense Agreement
Sublicense Agreement [Member]
Sublicense agreement.
Common Stock Warrants
Common Stock Warrants [Member]
Common stock warrants.
Related Party Payment of Contribution
Related Party Payment of Contribution
Contribution amount paid
Mereo BioPharma 3 Limited.
Mereo Bio Pharma3 Limited [Member]
Mereo
Asset Class
Asset Class [Axis]
Common stock, Change in fair value
Fair Value, Option, Changes in Fair Value, Gain (Loss)
Accruals of Research and Development Costs
Accruals Of Research And Development Costs Policy [Text Block]
Accruals of research and development costs.
Commitments And Contingencies Disclosure [Table]
Commitments And Contingencies Disclosure [Table]
Commitments and contingencies disclosure.
Deferred Tax Assets, Tax Credit Carryforwards, Total
Tax credits
Deferred Tax Assets, Tax Credit Carryforwards
Costs of property and equipment included in accounts payable, accrued, and other liabilities
Capital Expenditures Incurred but Not yet Paid
Total liabilities
Liabilities
Foreign rate differential
Effective Income Tax Rate Reconciliation, Foreign Income Tax Rate Differential, Percent
Schedule Of Property Plant And Equipment [Table]
Property, Plant and Equipment [Table]
Intangible assets, remaining amortization period
Finite-Lived Intangible Assets, Remaining Amortization Period
Finite lived intangible assets regulatory milestone period
Finite-Lived Intangible Asset, Useful Life
Intangible assets - Weighted Average Life
Interest receivable
Interest Receivable
Total stockholders’ equity
Beginning balance
Ending balance
Equity, Attributable to Parent
Acquired Finite-Lived Intangible Assets, Weighted Average Useful Life
Acquired Finite Lived Intangible Assets Weighted Average Useful Life
Upfront payment for exclusive option to acquisition
Business Combination Upfront Payment
Business combination upfront payment.
Daiichi Sankyo
Daiichi Sankyo Co Ltd [Member]
Daiichi Sankyo Co., Ltd.
Documents Incorporated by Reference [Text Block]
Documents Incorporated by Reference
Intangible assets, net
Intangible Assets, Net (Excluding Goodwill)
Intangible assets - Net Carrying Amount
Restricted Cash and Cash Equivalents, Current, Asset, Statement of Financial Position [Extensible List]
Restricted Cash and Cash Equivalents, Current, Statement of Financial Position [Extensible Enumeration]
Net loss
Net loss
Additional milestone payments.
Additional Milestone Payments
Additional Milestone Payments
Gain (loss) on securities held
Debt Securities, Held-to-Maturity, Derivative, Cumulative Gain (Loss)
Debt Securities, Held-to-Maturity, Derivative, Cumulative Gain (Loss), Total
Licenses Agreement [Member]
Licenses Agreement.
Licenses Agreement
Cost Sharing Payments [Table]
Cost Sharing Payments [Table]
Cost sharing payments table.
Collaborative Arrangement, Revenue Not from Contract with Customer, Statement of Income or Comprehensive Income [Extensible Enumeration]
Other
Other member.
Other Member
State income taxes, net of federal benefit
Effective Income Tax Rate Reconciliation, State and Local Income Taxes, Percent
Financing
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
Statement of Financial Position [Abstract]
Number of disease targets
Number Of Disease Targets
Number of disease targets.
Payments made under agreement.
Payments Made Under Agreement
Payments made under agreement
Shares used in computing net loss per share, Diluted
Weighted Average Number of Shares Outstanding, Diluted, Total
Weighted Average Number of Shares Outstanding, Diluted
Weighted-average shares used to computing net loss per share, diluted
RPI Finance Trust (RPI)
R P I Finance Trust [Member]
RPI Finance Trust.
Effective Tax Rate of Provision for Income Taxes from Federal Statutory Rate
Schedule of Effective Income Tax Rate Reconciliation [Table Text Block]
Stock price
Shares Issued, Price Per Share
Provision for income taxes
Effective Income Tax Rate Reconciliation, Percent
Federal
Deferred Federal Income Tax Expense (Benefit)
Aggregate purchase price
Payment For Purchase Of Common Stock
Payment for purchase of common stock.
Accruals and reserves
Deferred Tax Assets, Tax Deferred Expense, Reserves and Accruals
Deferred Tax Assets, Tax Deferred Expense, Reserves and Accruals, Total
Amortized Cost
Amortized Cost
Debt Securities, Available-for-Sale, Amortized Cost
Operating lease weighted average remaining lease term
Operating Lease, Weighted Average Remaining Lease Term
Percentage of statutory stock awards
Share-Based Compensation Arrangement by Share-Based Payment Award, Purchase Price of Common Stock, Percent
Use of Estimates
Use of Estimates, Policy [Policy Text Block]
Income Taxes
Income Tax Disclosure [Text Block]
Amount on adjustments for working capital and transaction expenses
Amount on Adjustments for Working Capital and Transaction Expenses
Amount on adjustments for working capital and transaction expenses
Deferred tax assets:
Deferred Tax Assets, Net [Abstract]
Weighted Average Exercise Price, Outstanding, Beginning Balance
Weighted Average Exercise Price, Outstanding, Ending Balance
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding, Weighted Average Exercise Price
2024
Operating And Finance Lease Liability Payments Due Next Twelve Months
Operating and finance lease liability payments due next twelve months.
Finite Lived Intangible Assets Line Items
Finite-Lived Intangible Assets [Line Items]
Computation of Basic and Diluted Net Loss per Share
Schedule of Earnings Per Share, Basic and Diluted [Table Text Block]
U.S. Government Treasury and Agency Securities
U S Government Treasury And Agency Securities [Member]
United states government treasury and agency securities.
Contractual payments received
Contractual Payments Received
Contractual payments received.
Disaggregation Of Revenue [Line Items]
Disaggregation of Revenue [Line Items]
Leasehold Improvements
Leasehold Improvements [Member]
Schedule of Changes in Fair Value of Investment in Securities
Schedule Of Changes In Fair Value Of Investment In Securities Table [Text Block]
Schedule of changes in fair value of investment in securities.
Income Tax Authority
Income Tax Authority [Axis]
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Warrants issued to purchase preferred stock
Stock and Warrants Issued During Period, Value, Preferred Stock and Warrants
Deferred Revenue Disclosure [Abstract]
Fair Value Disclosures [Abstract]
Total current tax provision
Current Income Tax Expense (Benefit)
Current state income tax expense
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax
Issuance Of Common Stock Upon Exercise Of Warrants And Under Equity Plan Awards Net Of Tax
Issuance of common stock upon exercise of warrants and under equity plan awards, net of tax
Level 2
Fair Value Hierarchy and NAV [Axis]
2026
Finance Lease, Liability, to be Paid, Year Three
Payments to Acquire Property, Plant, and Equipment, Total
Purchase of property, plant, and equipment
Payments to Acquire Property, Plant, and Equipment
Interest expense
Finance Lease, Interest Expense
Net proceeds from sale of future royalties
Proceeds from the sale of future royalties, net
Proceeds From Royalties Received Financing Activities
Proceeds from royalties received financing activities.
Milestone payments paid
Milestone Payments Paid
Milestone payments paid.
Share-Based Compensation Arrangement by Share-Based Payment Award, Equity Instruments Other than Options, Vested in Period
Number of shares, vested
GeneTx Biotherapeutics LLC
Gene Tx Biotherapeutics L L C [Member]
GeneTx Biotherapeutics LLC.
Common stock shares sold
Stock Issued During Period, Shares, New Issues
Other Commitments
Other Commitments [Axis]
Employee-related Liabilities, Current, Total
Payroll and related expenses
Employee-related Liabilities, Current
Commercial milestone payments
Commercial Milestone Payments
Commercial milestone payments.
Business Acquisition
Business Acquisition [Axis]
Total cash, cash equivalents, and restricted cash shown in the statements of cash flows
Cash, cash equivalents, and restricted cash at beginning of year
Cash, cash equivalents, and restricted cash at end of year
Cash, Cash Equivalents, Restricted Cash, and Restricted Cash Equivalents, Including Disposal Group and Discontinued Operations
2011 Equity Incentive, 2014 Incentive and Employment Inducement Plan
Two Thousand Eleven and Two Thousand Fourteen and Employment Inducement Plan [Member]
Two thousand eleven and two thousand fourteen and employment inducement plan.
Class of Warrant or Right
Class of Warrant or Right [Axis]
Income Tax Disclosure [Abstract]
Tax Credit Carryforward, Name
Tax Credit Carryforward, Name [Domain]
Finite-Lived Intangible Assets by Major Class
Finite-Lived Intangible Assets by Major Class [Axis]
Restricted Cash and Cash Equivalents, Noncurrent, Asset, Statement of Financial Position [Extensible List]
Restricted Cash and Cash Equivalents, Noncurrent, Statement of Financial Position [Extensible Enumeration]
North America
North America [Member]