Dr. Kakkis is best known for his work over the last 18 years to develop novel treatments for rare disorders. He began his work developing an enzyme replacement therapy (Aldurazyme®) for the rare disorder MPS I, with minimal funding and support. The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of the Ryan Foundation, a patient organization formed by Mark and Jeanne Dant for their son Ryan.
Aldurazyme development was later supported by BioMarin Pharmaceutical and eventually their partner Genzyme leading to FDA approval in 2003. During his tenure at BioMarin, Dr. Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU, and has contributed to the initiation of 7 other treatment programs for rare disorders, three of which are now in clinical development.
After 11 years at BioMarin, Dr. Kakkis left industry to initiate an effort to improve the regulatory and clinical development process for rare diseases. In early 2009, Dr. Kakkis launched and funded the Kakkis EveryLife Foundation to accelerate biotech innovation for rare diseases. The Foundation initiated a campaign to improve the regulatory and clinical development process for rare diseases. In just over a year, 160 patient organizations and physician society partners have endorsed the Campaign.
Dr. Kakkis has founded Ultragenyx™ to return to development of drugs for rare diseases. For many rare diseases, reasonable science exists that needs to get translated to patients. He will build on his previous experiences and will assemble an experienced team to efficiently develop treatments for rare diseases.
Dr. Kakkis is board certified in both Pediatrics and Medical Genetics. He graduated from Pomona College, magna cum laude and received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program and received the Bogen prize for his research. He completed a Pediatrics residency and Medical Genetics Training Fellowship at Harbor- UCLA Medical Center. He became an assistant professor of Pediatrics at Harbor-UCLA Medical Center from 1993 to 1998 where he initiated the enzyme therapy program for MPS I. In 1998, he joined BioMarin where he remained for 11 years in various titles eventually as Chief Medical Officer, before leaving in 2009.
Ms. Sharp joined Ultragenyx in May 2012 as Chief Financial Officer and Senior Vice President, Finance. She is a member of the Senior Management Team and is responsible for leading the corporate finance function, including strategic financial planning, accounting, budgeting and forecasting, financial analysis, general risk analysis, and financing activities.
Ms. Sharp is a member of the Board of Directors of Agenus Inc. (formerly Antigenics Inc.), a publicly traded biotechnology firm, where she served as Chief Financial Officer from 2006 to 2012. She joined Agenus in 2003 and held increasing roles of responsibility spanning strategic planning, corporate development, investor relations, corporate finance and business development. Prior to Agenus, Ms. Sharp held similar roles at Elan Pharmaceuticals from 1998 to 2003, including serving as chief of staff to the Chairman of the Board of Directors during that company's restructing. With 16 years of industry experience, Ms. Sharp has spearheaded numerous financing and business development transactions that have been critical to the success of Agenus and Elan. Prior to Elan, Ms. Sharp was a management consultant at McKinsey & Company as well as an investment banker at Goldman Sachs, specializing in pharmaceuticals and medical devices.
Ms. Sharp holds both a BA, magna cum laude, and MBA, from Harvard University
Steven Jungles brings more than 18 years of experience in supply chain and contract manufacturing. He joined Ultragenyx in August 2011 as Senior Vice President, Technical Operations, responsible for supply chain, manufacturing and quality control. Mr. Jungles worked at Somatix Therapy Corporation from 1993 to 1997, then was Associate Director of Operations at Harvard Gene Therapy Initiative in Boston, MA. In 1999, he met Dr. Emil Kakkis and began his career at BioMarin Pharmaceutical, where he held management and leadership roles of increasing responsibility, last holding the position of Vice President, Supply Chain and Contract Manufacturing. Mr. Jungles has a proven track record of successfully bringing four commercial products to market, including Aldurazyme, Naglazyme, Kuvan and Firdapse, and the development of multiple clinical products.
Mr. Jungles received his B.S. from the University of Iowa.
Mr. Ditton brings over 15 years of senior management background in strategic marketing and new product planning, with strong operational and sales knowledge developed as a leader of commercial teams in healthcare, rare diseases, pharmaceutical and medical device marketing. He has led successful U.S. and R.O.W. orphan product launches, in addition to serving in leadership roles with respect to marketing campaigns for large-market pharmaceutical products.
Mr. Ditton joined Ultragenyx in April 2011 and is responsible for commercial planning for pipeline products at the company. Mr. Ditton was most recently the Chief Operating Officer at EveryLife Foundation for Rare Diseases, a position he held from 2008 to 2011. Prior to Everylife Foundation, he served as the Vice President of Marketing at Diamics from 2006 to 2008, Director of Global Marketing at BioMarin Pharmaceutical from 2004 to 2006, Senior Product Manager at Merck/Dey from 2001 to 2004 and Business Development Manager at Health Plan of the Redwoods from 1994 to 2000.
Ms. Leonard heads up the company’s Regulatory Affairs department, whose members collectively have over 30 years of regulatory experience working with the FDA, EMA, PMDA, and other worldwide Regulatory Agencies. Approximately twenty of those 30 years she and her team spent working to develop drugs for Orphan indications at BioMarin Pharmaceutical. During her 8 year tenure at BioMarin, Ms. Leonard started up and led the CMC Regulatory group, then transferred to the Clinical/Nonclinical Regulatory group, where she advanced to lead that group as well. She was involved in securing various global approvals for Aldurazyme, Naglazyme, Kuvan and Orapred OPT. Prior to BioMarin, Ms. Leonard worked in Regulatory for 5 years at Cerus Corporation on drug/device combination products, in protein purification and analytical chemistry at Boehringer Mannheim, and in carbohydrate chemistry at Genentech.
Ms. Leonard received bachelor degrees in Chemistry and Biological Science from the University of California, Irvine and holds both US and EU Regulatory Affairs Certifications.
With more than 10 years of project and portfolio management experience from research to commercialization stage programs, Mr. Srivastava joined Ultragenyx in August 2011 and serves as Vice President, Program Development, overseeing the program development activities for the company. Mr. Srivastava’s experience and background includes over 20 years in the pharmaceutical and biotechnology industry, specifically focused on drug development in neurosciences and metabolic disorders including rare genetic disorders.
Before joining Ultragenyx, Mr. Srivastava was Senior Director, Portfolio and Project Management at Elan/Janssen Alzheimer Immunotherapy, a position he held from 2008 until 2011. He was also Director, Global Program Manager, Diabetes at Amgen from 2005 to 2008 and Director, Program Management at BioMarin Pharmaceutical from 2003 to 2005.
Mr. Srivastava holds a B.S. in Pharmacy from Banaras Hindu University, a M.S. in Medicinal Chemistry from St. Johns University and a M.A.S. in Management from Johns Hopkins University.
Dr. Vellard joined Ultragenyx as Vice President, Research in May 2013. Prior to joining Ultragenyx, Dr. Vellard worked as Head of Lysosomal Biology at BioMarin Pharmaceutical Inc., a biopharmaceutical company, from October 1999 to May 2013. He was a postdoctoral fellow in the pediatric department at UCLA Harbor Medical Center from September 1992 to June 1995.
Dr. Vellard received his B.S. in Natural and Life Sciences and M.S. in Molecular and Cellular Genetics from the University of Lyon I, France. He obtained his Ph.D. in Virology from the Pasteur and Curie Institutes (Universities Paris VI, VII and XI), France.
Dr. Cohrs has served as our Vice President of Quality since December 2013 and is responsible for overseeing our GMP, GLP, GCP, and Clinical Assay departments. Prior to Ultragenyx, Dr. Cohrs served as Senior Director of Quality Assurance/Quality Control at Medicines 360, a pharmaceutical company, from October 2012 through November 2013 and previously served as the Director of Quality Assurance at Medicines 360 from September 2009 through October 2012. Prior to Medicines 360, Dr. Cohrs served as the Director of Quality Assurance at Sunesis Pharmaceuticals Inc., a pharmaceutical company, from June 2007 to June 2008. Dr. Cohrs has also held increasingly responsible positions at a number of large pharmaceutical and small start-up companies, first in Quality Control and then in Quality Assurance, including Aventis Behring, Genetics Institute, Chiron, and InterMune. Dr. Cohrs has also contributed to numerous regulatory submissions resulting in several product approvals in both the United States and Europe. Dr. Cohrs received his Ph.D. in Organic Chemistry from Washington University and an M.B.A. in International Business from the University of Chicago.
Dr. San Martin has served as our Vice President, Clinical Development since December 2013. Prior to Ultragenyx, Dr. San Martin served as Senior Vice President of Clinical Development at Alder Biopharmaceuticals Inc., a pharmaceutical company, from January 2012 through May 2013. Prior to Alder, Dr. San Martin served as Executive Director and Global Development Leader at Amgen, Inc., a biotechnology company, from March 2006 to September 2011. Prior to Amgen, Dr. San Martin served as Medical Advisor at Eli Lilly and Company, a pharmaceutical company, from June 1998 to February 2006. Dr. San Martin received his M.D. from the University of Buenos Aires Medical School and completed his residence in internal medicine at CEMIC University of Buenos Aires.
Dr. Skrinar has served as the Senior Director, Clinical Sciences since March 2012. Prior to joining Ultragenyx, Ms. Skrinar worked as the Senior Director of Clinical Outcomes and Regulatory Affairs at Enobia Pharma, Inc., a private clinical stage orphan company acquired by Alexion in 2012 focused on the development of an enzyme replacement therapy for hypophosphastasia. Previously, as the Senior Director of Clinical Outcomes at Genzyme Corporation, she led the design and implementation of clinical trials of Myozyme/Lumizyme, an enzyme replacement therapy for Pompe disease and participated in the clinical development program for Aldurazyme, an enzyme replacement therapy for the treatment of MPS 1. In her nearly 15 years in biotech, Ms. Skrinar has worked exclusively on the clinical development and regulatory approval of ultra-orphan drugs.
Ms. Skrinar received a BBA from Emory University and a Ph.D. and MPH from the University of Alabama.
Spencer Guthrie brings over 15 years of clinical development experience in Biotech, in addition to several years of experience in Research, Project Management and Market Development Experience. Mr. Guthrie joined Ultragenyx In June 2012, and heads the Clinical Operations. Mr Guthrie’s experience include 8 years at Genentech with increasing responsibility culminating in overseeing many programs as a Sr. Clinical Program Manager. At Genentech, he worked on several innovative clinical programs, INDs and filings with Rituxan, Avastin and Lucentis, including work on orphan indications. Mr. Guthrie spent 5 years leading projects at Elan followed by Janssen Alzheimer’s Immunotherapy, working as Director of Clinical Operations, overseeing 6 compounds and a large group of employees, primarily in Neuroscience. He also served as the Project Management Lead for Tysabri and the Compound Core Team Leader for an early stage development project at Elan.
Mr. Guthrie has a Bachelor’s in Neuroscience from Vanderbilt University and an MBA from the University of California and is PMP certified.