8am – 5pm EST | 5am – 2pm PST

8:00 am Chair’s Opening Remarks

8:20 am Translating from Preclinical to Clinical in Duchenne’s

Synopsis

  • Exploring translatability of the dose response
  • Reviewing biodistribution with systemic delivery vs protein expression
  • Discussing construct design quality and expression levels

8:40 am Muscle Gene Therapy: How can we Effectively & Safely Express Transgenes in the Largest Body Compartment

  • Anne-Ulrike Trendelenburg Director, Head of MSD Cambridge, Novartis Institutes for Biomedical Research (NIBR), Musculoskeletal Disease Area (MSD), Novartis

Synopsis

  • Challenges with first generation approaches: Safety and efficacy due to high dose
  • Next generation capsids
  • Next generation payloads

9:00 am Q&A Panel Discussion

  • Carl Morris Chief Scientific Officer, Solid Bio
  • Anne-Ulrike Trendelenburg Director, Head of MSD Cambridge, Novartis Institutes for Biomedical Research (NIBR), Musculoskeletal Disease Area (MSD), Novartis
  • Michael Binks Vice President & Head of Rare Disease Clinical Research, RDRU, Worldwide Research & Development, Pfizer
  • Louise Rodino-Klapac Executive Vice President & Chief Scientific Officer, Sarepta Therapeutics

9:30 am Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:00 am Morning Break

Optimizing Gene Therapy Delivery with Next Generation Capsids & Muscle Specific Promoters

10:40 am Enhancing Muscle Targeting with Systemic Administration using Next Generation Capsids

  • Luk Vandenberghe Grousbeck Associate Professor in Gene Therapy, Harvard; Co-Founder & Chief Science Advisor, Affinia Therapeutics

Synopsis

  • AAV discovery requires multiparametric evaluation of key biodistribution, manufacturing and safety parameters
  • High-throughput AAVSeq approach allows for mouse and NHP screen to identify muscle-targeting and liver-detargeting AAVs
  • Rational design of commonly used AAV serotypes to detarget AAVs from liver with retained muscle tropism

11:00 am Optimizing the Selectivity of AAV for Target Muscle Cells Using Promoters

Synopsis

  • Identification of endogenous muscle-selective enhancer regions
  • Design of synthetic promoters utilising muscle enhancers
  • Characterisation of synthetic promoter activity in muscle groups in vivo

11:20 am Q&A Panel Discussion

  • Luk Vandenberghe Grousbeck Associate Professor in Gene Therapy, Harvard; Co-Founder & Chief Science Advisor, Affinia Therapeutics
  • Michael Roberts Chief Technology Officer, AskBio Europe

12:00 pm Lunch Break & Explore Platform

Clinically Meaningful Endpoints & Outcome Measures for Muscular Disorders

1:00 pm Beyond the 7 Minute Walk Test: Developing Clinically Meaningful & Objective Measures of Disease Improvement

  • Michael Binks Vice President & Head of Rare Disease Clinical Research, RDRU, Worldwide Research & Development, Pfizer

Synopsis

  • Talk details to follow

1:20 pm Roundtable Discussion: Seeking Translatable & Objective Biomarkers for Muscular Disorders

Synopsis

  • Progress in biochemical biomarkers
  • Implementing digital biomarkers
  • Advancing muscle biopsies

1:50 pm Developing AI-derived whole body movement fingerprints as clinical endpoints for DMD

  • Valeria Ricotti Co-founder, Exeutive Vice President & Chief Medical Officer, DiNAQOR AG

Synopsis

  • Conception of whole-body sensor-derived movement kinetics
  • Machine learning driven selection of disease features
  • Cross-sectional and longitudinal performance predictions compared to standard clinical assessments
  • Construction of a disease-life spanning ethomic fingerprints for DMD

2:10 pm Q&A Panel Discussion

  • Michael Binks Vice President & Head of Rare Disease Clinical Research, RDRU, Worldwide Research & Development, Pfizer
  • Carl Morris Chief Scientific Officer, Solid Bio
  • Valeria Ricotti Co-founder, Exeutive Vice President & Chief Medical Officer, DiNAQOR AG

2:30 pm Afternoon Break

Quality & Quantity: Addressing Manufacturing Challenges for Large Muscular Disorder Doses

2:50 pm Evaluating Non-Viral Targeted Delivery of Genes to Skeletal & Cardiac Muscle

Synopsis

  • Myosana Therapeutics has pioneered the development of non-viral gene delivery to skeletal and cardiac muscle
  • Our platform targets genes to muscle using an antibody to a unique target
  • The platform achieves systemic muscle expression of genes of any size

3:10 pm Q&A Panel Discussion: Manufacturing Challenges

Synopsis

  • From ophthalmic to muscular disorders: producing enough dose for systemic delivery & scaling up manufacture
  • Optimization of manufacturing processes to mitigate qc consequences on efficacy & toxicity

3:30 pm Chair’s Closing Remarks

3:45 pm Scientific Poster Session

4:45 pm End of Day