TRANSLATION & REVERSE TRANSLATION: DOSAGE & LONG TERM FOLLOW UP

7:45 am Morning Refreshments

8:00 am Targeting Cardiac Muscle

  • Roger Hajjar Therapeutic Sector Leader, Cardiac Gene Therapy, AskBio

Synopsis

  • Cardiac specific delivery of vectors has been achieved using minimally invasive techniques clinically
  • AAV vectors have been used safely in early cardiac gene therapies, but myocardial uptake has been inefficient so far
  • Novel re-engineered AAV vectors are increasing the efficiency of myocardial uptake
  • The scope of cardiac diseases that is being targeted by gene therapy is expanding from monogenic to acquired diseases

8:30 am An Immune Evading Vectors for Treating Genetic Diseases

Synopsis

  • Review of current safety challenges facing AAV based gene therapies
  • Introducing the EVADER platform, which results in products that are more potent and less immunogenic
  • Exploring the advantages for treating genetic diseases affecting muscle cells

9:00 am Optimizing Long Term Follow Up Strategies & Data Collection for Future Muscular Disorder Studies

Synopsis

  • 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 therapy recipients

9:30 am Morning Break

10:29 am
UNDERSTANDING MUSCLE BIOLOGY TO OPTIMIZE THERAPEUTIC INTERVENTION

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

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

Synopsis

  • 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 responses

11:00 am Translating AAV8-Microdystrophin Research in Dogs, Rats & Humans

Synopsis

  • Assessing safety and efficacy preclinical data in GRMD dogs and DMD-rats
  • Long term efficacy and safety data in the animal models
  • Clinical development plan and first data
  • Discussion on immunological, cardiac and skeletal muscle issues

11:30 am Evaluating Muscle Turnover & Discussing Satellite Cell Targeting for Improved Durability

Synopsis

  • Talk details to follow

12:00 pm Discovering a Novel Capsid & Promoter for LGMDs

Synopsis

  • Talk details to follow

12:30 pm Lunch & Networking

1:29 pm
DELVING DEEPER INTO IMMUNOGENIC RESPONSES

1:30 pm Addressing Anti-AAV Immune Responses Preclinically

  • Helene Haegel Immunosafety Scientist, Pharmaceutical Sciences, Roche Pharma Research & Early Development, Roche Innovation Center Schlieren, Roche

Synopsis

  • Immunogenicity risk assessment
  • Exploring immunogenicity mitigation strategies

2:00 pm Avoiding a Problem Doesn’t Solve it; Overcoming Pre-existing Anti-AAV Neutralizing Antibodies

Synopsis

  • Pre-existing anti-AAV neutralizing antibodies (NAbs) preclude some patients from receiving systemic AAV gene therapy
  • Vector readministration is also currently impossible in patients who only realize suboptimal transgene expression due to high NAb from the first vector administration
  • Emerging strategies that reduce or eliminate NAbs can increase patients treated with AAV while allowing vector readministration

2:30 pm Roundtable Discussion: Muscular Biomarkers & Outcome Measures

At the first in-person Gene Therapy for Muscular Disorders event, maximize your networking opportunities by sharing your research and exploring your peers posters to improve your understanding of the field and make new connections.

1. Optimizing Biomarker Development

2. Immunogenicity & Safety

3. Defining Clinical Endpoints

3:15 pm Afternoon Break

ADVANCING GENE THERAPY RESEARCH

3:45 pm Enhancing Collaborations & Standardization for Muscular Disorders

Synopsis

  • 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 targets

4:15 pm Panel Discussion: Unifying Muscular Disorders Stakeholders Through Collaborations & Standardizations

Synopsis

  • 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 studies

5:00 pm Chairs Closing Remarks

5:15 pm End of Conference