Pre-Conference Workshop Day

Monday, April 4 2022

WORKSHOP A: Gene Therapy for Cardiovascular Disorders: Targeting Cardiac Muscle

This workshop will evaluate gene therapy as a therapeutic modality for targeting cardiac disorders including hypertrophic cardiomyopathy (HCM) and arrhythmogenic right ventricular cardiomyopathy.

This workshop will cover:

  • Exploring inherited diseases of the heart muscle
  • Showcasing preclinical and clinical progress with gene therapies

Workshop Leader:

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Jay Barth
Chief Medical Officer
Lexeo Therapeutics

WORKSHOP B: Developing a Biomarker Safety Package

Evaluate the utility of multiple safety biomarkers to evaluate gene therapy safety for muscular disorders, including liver and cardiac safety biomarkers.

This workshop will cover:

  • Which biomarkers are specifically required for a biomarker safety package for muscular disorders patient populations?
  • What needs to be characterized for different AAV vectors, such as DRG toxicity?
  • Addressing sensitivity and reliability challenges
  • Discussing what is required by health authorities and regulators

Workshop Leader:

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Laurence Whiteley
Senior Director

WORKSHOP C: Gene Editing for Muscular Disorders

CRISPR gene editing has provided new opportunities to ameliorate the disease by eliminating DMD mutations and thereby restore dystrophin expression throughout skeletal and cardiac muscle. Proof-of-concept studies in rodents, large mammals, and human cells have validated the potential of this approach.

This workshop will discuss:

  • Gene editing approaches for treating muscular disorders
  • The challenges and opportunities facing gene editing
  • Optimization of gene editing
  • Delivery of gene editing components throughout the musculature
  • Mitigation of possible immune responses
  • Translation into other muscular disorders

Workshop Leader:

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Tudor Fulga
Vice President, Gene Editing
Vertex Cell & Genetic Therapies