Pre-Conference Workshop Day

Monday, April 4 2022

WORKSHOP A: Gene Therapy for Cardiovascular Disorders: Targeting Cardiac Muscle
8AM-11AM

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:

REW Pardot speaker photos (3)

Jay Barth
Chief Medical Officer
Lexeo Therapeutics

WORKSHOP B: Developing a Biomarker Safety Package
11.30AM-2.30PM

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
Pfizer

WORKSHOP C: Gene Editing for Muscular Disorders
3PM-6PM

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