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Congressional Briefing: Realizing the Promise of Gene Therapy through Science & Policy

On February 21, the Institute for Gene Therapies hosted a panel discussion on the science of gene therapy, its impact on patients and families, and the role of Congress in helping to ensure patient can benefit from these transformational therapies. IGT Chairman Erik Paulsen moderated the discussion with the following speakers:

  • Sudha Sharma, Ph.D. National Genome Center at Howard University and Member of IGT's Scientific, Academic, & Medical Advisory Council

  • Nate Plasman, whose son is enrolled in a gene therapy for Duchenne muscular dystrophy

  • John Feore, IGT’s Director of Policy

Watch the discussion here:

Dr. Sudha Sharma spoke to attendees about the unique nature of gene therapies, the progress in the field in recent years, including the diseases gene therapy can treat and what is on the horizon, and how gene therapies may help address health disparities.

“There has been tremendous recent advancement in gene therapy. There is an urgent need to match this momentum with healthcare and regulatory policies to make gene therapies accessible to a diverse patient population.” – Dr. Sudha Sharma

Nate Plasman gave compelling remarks about his son Andrew, who has Duchenne muscular dystrophy. Since 2019, Andrew has been in a clinical trial for a gene therapy developed by IGT Corporate Advisory Council member Sarepta Therapeutics. The gene therapy is currently under review by the FDA for accelerated approval. Mr. Plasman spoke about the experience of Andrew participating in the clinical trial and the profound impact that gene therapy has had on his son and his whole family.

“My son will be able to walk across the stage and receive his high school diploma. We envision him going to college, having a career, falling in love and having a family, which is a paradigm shift for people with Duchenne muscular dystrophy because of gene therapy. A new day is dawning.” – Nate Plasman

John Feore highlighted the policy changes and actions by Congress that are needed to address the barriers patients may face accessing these transformative therapies.

IGT will host additional panel discussions throughout the year and serve as a resource for stakeholders on the progress in the field and the policy implications of these life-changing treatments.


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