AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy - 2021 World Medical Innovation Forum
Virtual
May 19–21, 2021

AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy

AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy

Wednesday, May 19, 2021

10:20 AM – 10:45 AM

Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.

The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.

  • Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
  • Lessons learned from these first-in-class approvals.
  • Challenges to broaden this modality to similar indications.
  • Reflections on safety signals in the clinical studies?
Moderator:
Joan Miller, MD
  • Chief, Ophthalmology, MEE
  • Cogan Professor & Chair of Ophthalmology, HMS
Speakers:
Ken Mills
  • CEO, RegenXBio
Eric Pierce, MD, PhD
  • Director, Ocular Genomics Institute, MEE
  • Professor of Ophthalmology, HMS
Ron Philip
  • Chief Operating Officer, Spark Therapeutics
Meredith Schultz, MD
  • Executive Medical Director, Lead TME, Novartis Gene Therapies
  • Q&A

    10:50 AM – 11:05 AM

the Future of Medicine and AI

May 11–13, 2020 | Boston, MA