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The do’s and don’ts of managing a pivotal gene therapy trial

Gene therapies have been exploding in popularity, with big and small players lining up scores of new programs looking to create entirely new standard of care models for a wide variety of diseases. But what does it take to de-risk a pivotal, registration-worthy gene therapy program? What are the pitfalls to avoid and the opportunities in creating more efficient designs that are more likely to get a nod from regulators?

Join Deborah Wild, Chief of Staff and VP of Corporate Development at Catalent; Kathy Reape, Chief Medical Officer at Spark Therapeutics; Bobby Gaspar, President of Research and Chief Scientific Officer at Orchard Therapeutics; Michael Binks, Vice President of Clinical Research at Pfizer; Wing Yen Wong, Vice President of Clinical Science at BioMarin; and Endpoints News editor John Carroll as they discuss this and more during our upcoming webinar.

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Deborah Wild

Deborah Wild

Chief of Staff & VP of Corporate Development, Catalent

Deborah Wild is an accomplished Quality and executive leader with more than 30 years of experience. Most recently she ran a thriving consulting practice for more than 14 years where she lead Quality Assurance focused initiatives for high profile clients such as Genentech, TEVA, Polynoma, Cytovance, and CMC Biologics. While simultaneously managing her consulting business, Ms. Wild occupied roles at Polynoma as VP of Quality Systems and VP of Manufacturing and Supply Chain at Victory Pharma. Prior to these roles, she spent more than 17 years at Genentech, with her final title as Director of Contract Manufacturing.

Through her experience, Ms. Wild has earned a strong reputation for adding to company growth strategies by effectively transforming Quality organizations into strong business partners. Additionally, Ms. Wild was a member of the United States Army and was deployed to Desert Storm toward the end of her enlistment. She began as a Private First Class and left having achieved the rank of Staff Sergeant.

Kathy Reape

Kathy Reape

Chief Medical Officer, Spark Therapeutics

Kathy Reape is the Chief Medical Officer of Spark Therapeutics and oversees clinical, pharmacovigilance and medical affairs activities. With more than 20 years of pharmaceutical industry experience in clinical drug development, Kathy has overseen numerous clinical trials and had a hands-on role in the approvals of more than two dozen products including small molecules, biologics, biosimilars, and devices spanning a wide range of therapeutic areas.

Prior to joining Spark, Kathy was the president of Ark Medical Consulting LLC where she provided pharmaceutical and medical consulting and the Senior Vice President, Head of Clinical Development, Global Brands R&D for Allergan, plc where she led the Clinical Development function for the entire brand R&D portfolio, including projects in ophthalmology, cardiovascular, gastrointestinal, anti-infectives, respiratory, women’s health, urology, dermatology/aesthetics, and central nervous system (CNS). She also oversaw all development work related to Allergan’s onabotulinumtoxinA (BOTOX®). Kathy has also held leadership positions at Teva Pharmaceuticals, Duramed Research and Wyeth Pharmaceuticals.

She received her M.D. from the University of Pennsylvania and completed her internship and residency at the University of Florida and the University of Medicine and Dentistry of New Jersey.

Wing Yen Wong

Wing Yen Wong

Vice President of Clinical Science, BioMarin

Michael Binks

Michael Binks

Vice President Clinical Research, Pfizer

Michael Binks joined Pfizer in 2014 to lead Clinical Research within the Rare Diseases Research Unit. He is currently the research project leader for the DMD gene therapy program. He qualified in medicine at University College London (UCL), UK before training in internal medicine, gastroenterology, neurology, respiratory and emergency medicine and rheumatology.

Michael Binks is an UK accredited Rheumatologist. My scientific interests have been in immunology and immunopharmacology. He served as a Lecturer in Immunology and as an Arthritis Research Council and Jules Thorn Clinical Fellow at UCL, conducting research on antigen presentation and dendritic cell biology including the rare diseases Wiskott Aldrich Syndrome and scleroderma.

He joined the pharmaceutical industry in immune-inflammation discovery biology, translational and experimental medicine and clinical pharmacology and have progressed programs from discovery to the clinic to POC.

He is the author of over 40 peer reviewed publications and 1 patent.

Bobby Gaspar

Bobby Gaspar

President of Research and Chief Scientific Officer, Orchard Therapeutics

Bobby is a professor of pediatrics and immunology at the UCL Great Ormond Street Institute of Child Health. He has a special interest in the treatment of severe primary immune deficiencies, including bone marrow transplantation and gene and cell therapy. Bobby has led multiple clinical trials that have shown that gene therapy can successfully correct the genetic defect in immune deficiencies. Bobby has led UK and European-wide initiatives for newborn screening in severe combined immune deficiency.

John Carroll
moderator

John Carroll

Founder & editor, Endpoints News

John D. Carroll is a biotech analyst and writer with decades of prize-winning experience in journalism. A co-founder of Endpoints News, he has covered biopharma for the past 15 years. Aside from his previous daily industry coverage at FierceBiotech, where he was named editor in 2003, leaving in 2016, Carroll has been a regular speaker at biotech events around the globe. He’s been quoted by The New York Times, The New Yorker, Financial Times, The Times of London and more. He’s also keynoted at biotech gatherings around the world and addressed student audiences at MIT and Harvard.