Sharon Hesterlee

Sharon Hesterlee

Company: Muscular Dystrophy Association

Job title: Chief Research Officer


Panel Discussion: Unifying Muscular Disorders Stakeholders Through Collaborations & Standardizations 2:30 pm

Sharing the perspectives of patients who have enrolled in gene therapy trials Discussing the importance of patient-centric trials Feeding back what industry developers can improve upon for future studiesRead more

day: Day Two

Strategies for Gene Therapy in indications with low commercial viability 2:00 pm

The majority of neuromuscular diseases in MDA’s program affect less than 1000 people in the United States To better address the needs of people with genetic NMDs that are not commercially viable MDA is launching an in-house biotech incubator to de-risk gene therapy programs for technologically feasible targetsRead more

day: Day Two

Panel Discussion: Evaluating Different Gene Editing Approaches for Muscular Disorders: Challenges & Opportunities 10:30 am

Highlighting the positive preclinical results of genetic expression in the muscle using gene editing Evaluating different gene editing approaches for muscular disorders Exploring delivery of gene editing components throughout the musculature Discussing the safety considerations for gene editing approaches and mitigation of possible immune responsesRead more

day: Day Two

Optimizing Long Term Follow Up Strategies & Data Collection for Future Muscular Disorder Studies 9:10 am

The requirements for long-term follow up of gene therapy trial participants and for those who receive an approved gene therapy add complication and cost to the development of these therapies The Muscular Dystrophy Association’s Care Center Network and MOVR database are being positioned to help meet the needs for administration and long-term follow-up of gene…Read more

day: Day Two

Panel Discussion: Overcoming Manufacturing Challenges with Advancing Programs 4:15 pm

Discussing the additional potency assays requested by regulators Debating the affect of quality on efficacy Approaching later programs and ensuring quality scale up and avoiding bottlenecks with large doses required for high muscle massRead more

day: Day One

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