5th Gene Therapy for Muscular Disorders

5th Gene Therapy for Muscular Disorders is a conference dedicated to genetic therapies targeting muscular disorders, including Limb-Girdle Muscular Dystrophy (LGMD), Duchenne Muscular Dystrophy (DMD), X-Linked Myotubular Myopathy and Pompe Disease.

Key Topics

• Muscle Gene Therapy: How can we Effectively & Safely Express Transgenes in the Largest Body Compartment
• Translating from Preclinical to Clinical in Duchenne’s
• Clinically Meaningful Endpoints & Outcome Measures for Muscular Disorders
• Optimizing Gene Therapy Delivery with Next Generation Capsids & Muscle Specific Promoters
• Exploring CRISPR-based Gene Editing for Muscular Disorders
• Quality & Quantity: Addressing Manufacturing Challenges for Large Muscular Disorder Doses
• Preclinical Model Challenges to Effectively Determine Dose
• Lessons Learned: Mitigating Toxicity & Immunogenicity Challenges
• Clinical Challenges for Muscular Disorders