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Gene Therapy – A New Treatment Modality: Opportunities and Challenges from a Patient’s, Industry, Regulatory and Payer’s Perspective
Session Chair(s)
Sam Wadsworth, PhD
Chief Scientific Officer, Dimension Therapeutics, United States
Modifying cellular gene expression to treat diseases has been a long-term goal which, due to novel technologies, now seems to be reachable. The panel will discuss emerging opportunities and hurdles that need to be overcome to make this dream a reality.
Speaker(s)
Marcel Hoefnagel, DRSc, PhD, MSc
Senior Assessor Biopharmaceuticals, Medicines Evaluation Board , Netherlands
Gene Therapy- A New Treatment Modality- Opportunities and Challenges from a Regulatory Perspective
Dimitrios Athanasiou, MBA
Patient Advocate , WORLD DUCHENNE ORGANIZATION (WDO) , MDA HELLAS, Greece
Gene Therapy – The Patient’s Perspective
Deya Corzo
SVP, Therapeutic Area Head, UniQure, United States
Challenges in Bringing New Treatment Modalities to Patients
Anne-Virginie L. Eggimann, MS
Chief Regulatory Offcer, bluebird bio, Inc., United States
Adaptive Biomedical Innovation – A Supportive Framework for Accelerating the Development of Gene Therapy Products
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