7:59 am
DISCUSSING STRATEGIES TO SOLVE THE ADVERSE CLINICAL EFFECTS OF LARGE DOSES FOR MUSCULAR TARGETS

8:00 am Morning Refreshments

8:30 am Chair’s Opening Remarks

8:45 am A Clinician’s Perspective: Gene Therapy for X-Linked Myotubular Myopathy

  • Perry Shieh Professor of Neurology & Pediatrics, University of California Los Angeles & Principal Investigator for ASPIRO, Astellas Gene Therapies

Synopsis

  • -X-Linked Myotubular Myopathy (XLMTM): Burden of disease
  • Gene therapy and efficacy outcomes
  • Safety profile and liver toxicity

9:15 am Panel Discussion: The Race for A Cure: Field Update of Muscular Disorders Gene Therapy Programs

  • Anh Nguyen Head of Limb Girdle Muscular Dystrophy, AskBio
  • Carl Morris Chief Scientific Officer, Solid Biosciences Inc.
  • Perry Shieh Professor of Neurology & Pediatrics, University of California Los Angeles & Principal Investigator for ASPIRO, Astellas Gene Therapies
  • Louise Rodino-Klapac Chief Scientific Officer, Sarepta Therapeutics

Synopsis

  • Addressing older patient populations & introducing even larger doses
  • Clinicians managing the adverse effects: myocarditis and liver toxicity
  • Discussing predisposition to adverse side effects

10:00 am Structured Networking

11:00 am Morning Break

ASSESSING THERAPEUTIC EFFICACY & SAFETY IN MUSCULAR DISORDERS

11:30 am Investigating Clinical Data & Improving Monitoring with Novel Assays

Synopsis

  • Anti-capsid and anti-transgene antibody characterization with novel multiplex assays
  • Isotyping of anti-capsid antibodies
  • Domain mapping of anti-transgene protein antibodies

12:00 pm Utilizing Preclinical Models to Demonstrate Efficacy & Durability for Muscular Disorders

  • Marla Weetall Senior Vice President, Pharmacology & Biomarkers, PTC Therapeutics

Synopsis

  • Challenges in mouse models of DMD and in measuring dystrophin
  • Use of corticosteroids in mouse models of DMD and in the clinic
  • Measuring dystrophin increases in mouse models of DMD and in the clinic

12:30 pm Lunch & Networking

1:59 pm
EXPLORING NOVEL IN VITRO & IN VIVO PRECLINICAL ANIMAL MODELS OF MUSCULAR DISORDERS

2:00 pm Utilizing Multiple Animal Models to Evaluate Therapeutic Efficacy

Synopsis

  • Gene therapy studies in small animal models
  • Functional efficacy evaluations
  • Utility of canine models for further evaluation

2:30 pm Utilizing In Vitro 3D Muscle Bundles to Assess the Functionality of AAV Therapeutics for Duchenne Muscular Dystrophy (DMD)

Synopsis

  • Reproducible assay to test gene therapy candidates in DMD-patient derived cells
  • Repair of truncated dystrophin at the sarcolemma of DMD-treated myotubes
  • Structural restoration of the DAPC complex in DMD myotubes treated with an AAV-mediated exon skipping

3:00 pm Afternoon Break

3:30 pm Patient’s Perspective: Receiving a Gene Therapy & Understanding What is Critical for Patients

Synopsis

  • Talk details to follow

NAVIGATING REGULATORY & MANUFACTURING CHALLENGES FOR MUSCULAR DISORDERS

4:00 pm Case Study: Scaling Up & Manufacturing Large Quantities for Large Target Organ

  • Chris Lorenz Senior Vice President, Technical Operations, Astellas Gene Therapies

Synopsis

  • Walking through the manufacturing challenges faced with programs entering later phases
  • Discussing strategies used to navigate these challenges
  • Suggested considerations for future gene therapy trials for muscular disorders

4:30 pm Approaching Regulators & Navigating Regulatory Challenges

Synopsis

  • Overview of CMC, nonclinical and clinical regulatory challenges
  • Planning for success
  • Engaging regulators

5:00 pm Panel Discussion: Overcoming Manufacturing Challenges with Advancing Programs

Synopsis

  • Discussing the additional potency assays requested by regulators
  • Debating the affect of quality on efficacy
  • Approaching later programs and ensuring quality scale up and avoiding bottlenecks with large doses required for high muscle mass

5:30 pm Chairs Closing Remarks

5:45 pm Scientific Poster Session

Synopsis

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.

7:00 pm End of Conference Day One