9am – 4pm EST | 6am – 1pm PST

Future Considerations For Optimal Genetic Therapies

8:40 am Exploring CRISPR-based Gene Editing for Muscular Disorders

  • Alison McVie-Wylie Vice President, Disease Area Executive, DMD, Vertex Cell & Genetic Therapies


  • Review the current status of gene editing research and development for muscle diseases
  • Discuss the differentiators to gene replacement
  • Present potential mitigation strategies to limit off-target effects

9:00 am Determining Gene Therapy Efficacy with Preclinical Models


  • Talk details to follow

9:20 am Common Challenges Faced in Developing Gene Therapies for Neuromuscular Diseases

  • Serge Braun Director of Neuromuscular Strategy, Genethon


Talk details to follow

9:40 am Q&A Panel Discussion: Assessing Different Approaches for DMD

  • Alison McVie-Wylie Vice President, Disease Area Executive, DMD, Vertex Cell & Genetic Therapies
  • Marla Weetall Vice President, Pharmacology, PTC Therapeutics
  • Serge Braun Director of Neuromuscular Strategy, Genethon

10:10 am Morning Break

Lessons Learned: Mitigating Toxicity & Immunogenicity Challenges

10:30 am Understanding the Immunogenic Response: Complement Activation, Transgene & Vector Responses


  • The criticality of immunogenicity evaluation for AAV-based gene therapies
  • Methods for detecting anti-capsid binding antibodies, neutralizing antibodies and anti-gene product antibodies
  • Experience implementing these assays for a gene therapy for Duchenne Muscular Dystrophy
  • A novel approach to isotyping of anti-capsid antibodies, and the potential relevance of isotype on adverse events such as complement activation

10:50 am Optimizing Post Treatment Patient Monitoring Strategies for Gene Therapy Safety

  • Mark Milton Executive Director, PK Sciences, Novartis


  • What are the commonly observed safety signals for AAV gene therapies?
  • Exploring the risk mitigation strategies that can be applied
  • What is the right balance between collecting safety data and the patient experience?

11:10 am Exploring Redosing Strategies for Muscular Disorders: Mitigation of AAV Immunogenicity with ImmTOR Tolerogenic Nanoparticles


  • ImmTOR is a clinically validated platform technology that has been shown to selectively mitigate unwanted immunogenicity of biologic
  • Preclinical studies have demonstrated the ability to inhibit anti-AAV antibody responses and enable vector re-dosing
  • ImmTOR also has a benefit of enhancing transgene expression at the first dose
  • Presenting data from a recent NHP study

11:30 am Q&A Panel Discussion: Mitigating Toxicity

12:00 pm Lunch Break & Private Calls

Harnessing the Value of Preclinical Model Challenges to Effectively Determine Dose

1:00 pm Utilizing Canine Models to Study DMD Gene Therapy


  • Exploring the advances in DMD canine models
  • Application of canine DMD models to gene replacement and gene repair therapy
  • Highlighting the benefits of canine vs other animal models

1:20 pm The Role of Biomarkers in Understanding Muscular Dystrophy

1:40 pm Q&A Panel Discussion: Overcoming Translational Challenges

1:50 pm Afternoon Break

Navigating Clinical Challenges for Muscular Disorders

2:40 pm Highlighting the Importance of Collaborations to Propel Gene Therapy Success in Muscular Disorders


  • Highlighting the power of collaboration for muscular disorders research
  • Discussing ultra-rare muscle disease and the intersection between nonprofits, individual families and industry to make some of these disorders more viable commercial targets for gene therapy
  • Understanding logistical issues, ethical considerations and reimbursement considerations

3:00 pm Considerations for Industry Developers with Trial Design from a Patient’s Perspective

  • Pat Furlong Founding President & Chief Executive Officer, Parent Project Muscular Dystrophy


  • Highlighting the value of patient/caregiver input in the development of gene therapy protocols
  • Seeking clinically relevant endpoints that are meaningful to patients and integrating real world evidence (RWE) to inform regulatory decisions

3:20 pm Q&A & Panel Discussion: Collaborations for Future Research

  • Pat Furlong Founding President & Chief Executive Officer, Parent Project Muscular Dystrophy
  • Sharon Hesterlee Chief Research Officer, Muscular Dystrophy Association
  • Debra Miller CEO & Founder, CureDuchenne

3:50 pm Chair’s Closing Remarks

4:00 pm End of Conference