Program

The interactive Breakthroughs in Muscular Dystrophy (November 19-20, 2024) program below is updated live as speakers and sessions are confirmed.

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Program

Tuesday, November 19

Keynote - Genetic Therapies for Muscular Dystrophies: Opportunities and Challenges
8:35 - 9:35 a.m.
Chair: Jeffrey Chamberlain, PhD, University of Washington

• Genetic Therapies for Muscular Dystrophies: Opportunities and Challenges
  Louis Kunkel PhD, Children's Hospital Boston
• Genetic Therapies for Muscular Dystrophies: Opportunities and Challenges
  Carsten Bonnemann MD, NINDS/NIH

Non-Viral Delivery
9:35 - 10:35 a.m.
Chair: Monkol Lek, PhD, Yale School of Medicine

• Advancing Non-Viral Delivery via Battelle's HIT SCAN Platform
  Kenneth Sims PhD, Battelle Memorial Institute
• A targeted, non-viral platform to deliver the full-length dystrophin gene
  Nick Whitehead PhD, Myosana Therapeutics

Viral Delivery
11:05 a.m. - 12:05 p.m.
Chair: Melissa Spencer, UCLA

• Monitoring Viral Delivery with Non-invasive Imaging
  Oluwatayo Ikotun, University of California, Los Angeles
• Split-Inteins
  Jeffrey Chamberlain PhD, University of Washington

Monitoring AAV Delivery
1:05 - 2:30 p.m.
Chair: Carsten Bonnemann, MD, NINDS/NIH

• Intro to Monitoring AAV Delivery
  Federico Mingozzi PhD, Spark Therapeutics Inc

• Vector Immunology
  J. Fraser Wright PhD, Stanford School of Medicine

• Transgene Immunology
  Armando Villalta PhD, UC Irvine

• Enabling Readministration/Redosing
  Barry Byrne MD, PhD, University of Florida

Gene Editing
2:40 - 4:00 p.m.
Chair: Angela Lek, VP of Research, MDA

• A Novel Dual Vector Approach for Safe and Efficient Expression of Large Genes using RNA End-joining (REJ)
  Samuel Pfaff PhD, The Salk Institute for Biological Studies
• Genome Editing for Duchenne Muscular Dystrophy
  Charles Gersbach PhD, Duke University
• Panel Discussion

Oral Abstract Session
4:30 - 5:50 p.m.
Chairs: Evrim Atas and Angela Lek, MDA

Wenesday, Novemeber 20

RNA Targeted Therapies
8:30 - 10:15 a.m.
Chair: Scott Harper, Ohio State University & Nationwide Children's Hospital

• Enhancing the Delivery of Antisense Oligonucleotide Therapies: Innovative Approaches for Muscular Dystrophy
  Toshifumi Yokota PhD, University of Alberta, Edmonton, AB
• Translating RNAi-based Gene Therapies for Dominant Neuromuscular Disorders
  Lindsay Wallace PhD, Nationwide Children's Research Institute, Columbus, OH
• ASOs for CNS delivery
  Aurelie Goyenvalle PhD, University of Versailles Saint Quentin En Yvelines
• Panel Discussion

Panel: Future of Genetic Therapies for Muscular Dystrophies
10:45 a.m. - 12 p.m.
Chair: Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association

• Panelists
  Jeffrey Chamberlain PhD, University of Washington
  Carsten Bonnemann MD, NINDS/NIH
  Katherine High MD, Rockefeller University
  Barry Byrne MD, PhD, University of Florida
  Melissa Spencer, UCLA

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Thank You to the Organizing Committee

• Jeffrey Chamberlain, PhD
  University of Washington
• Angela Lek, PhD
  Muscular Dystrophy Association
• Evrim Atas, PhD
  Muscular Dystrophy Association
• Charlie Gersbach, PhD
  Duke University
• Toshifumi Yokota, PhD
  University of Alberta
• Manuela Corti, PhD
  University of Florida
• Monkol Lek, PhD
  Yale University
• Julie Crudele, PhD
  University of Washington
• Melissa Spencer, PhD
  UCLA
• Carsten Bonnemann, MD
  NIH
• Scott Harper, PhD
  The Ohio State University/Nationwide Children's Hospital