1, Avenue

De Lafayette Boston,

MA,

USA,

ZIP: 02111

Phone: +16174554188

The 2nd Gene Therapy for Muscular Disorders Summit will be returning as an in-person event to Boston in April 2022, to help gene therapy developers targeting muscular disorders to overcome safety and toxicity challenges and effectively translate their programs into the clinic.

Highlighting AAV and gene editing challenges for Duchenne's (DMD), Limb-Girdle (LGMD), Pompe Disease, X-Linked Myotubular Myopathy and other muscular disorders, this industry-focused 4-day event will cover:

-Overcoming immunogenicity challenges and mitigate toxicity with high doses
-Optimizing vector and promoter choice and design for more efficient delivery tomuscle targets
-Defining clinically meaningful muscular endpoints to successfully develop and safely deliver the optimal dose to their muscular targets

Join over 150 fellow industry experts to learn, share and benchmark yourselves against your competitors, and overcome the toxicity and dosage challenges hindering the progress of your gene therapy targeting the muscle.

Group discounts and early booking rates are available. Please visit the website for full pricing information.

URLs:
Brochure: https://go.evvnt.com/944879-2?pid=1052
Tickets: https://go.evvnt.com/944879-3?pid=1052

Alison McVie-Wylie, Vice President, Disease Area Executive, DMD, Vertex Cell and Genetic Therapies, Anh Nguyen, Vice President, Therapeutic Sector Lead - Musculoskeletal, AskBio, Carl Morris, Chief Scientific Officer, Solid Biosciences Inc., Chris Lorenz, Senior Vice President, Technical Operations, Astellas Gene Therapies, Genine Winslow, Chief Scientific Officer, Chameleon Bioscience, Helene Haegel, Immunosafety Scientist, Pharmaceutical Sciences, Roche Pharma Research and Early Development, Roche Innovation Center Schlieren, Roche, Jay Barth, Chief Medical Officer, Lexeo Therapeutics, Jennifer Green, Principal Scientist, R and D, Solid Biosciences, Jennifer Levy, Scientific Director Coalition to Cure, Calpain 3 (C3), Jose Trevejo, Officer and Senior Vice President of AAV, Rocket Pharmaceuticals, Joseph Rabinowitz, Joseph Rabinowitz, Kriya Therapeutics, Juan Manuel Iglesias, Director of Product R and D, Europe, Europe AskBio, Laurence Whiteley, Senior Director, Pfizer, Lianna Orlando, Senior Director of Research, CureDuchenne, Lisa Dyleski, Senior Scientist, Bioanalysi, Pfizer, Louise Rodino-Klapac, Chief Scientific Officer, Sarepta Therapeutics, Maritza McIntyre, Chief Development Officer, Stride Bio, Marla Weetall, Senior Vice President, Pharmacology and Biomarkers, PTC Therapeutics, Melissa Kotterman, Chief Scientific Officer, Iris Medicine, Olivier Danos, Chief Scientific Officer. REGENXBIO, Pat Gonzalez, Associate Director, R and D, Solid Biosciences, Perry Shieh, Professor of Neurology and Pediatrics, University of California Los Angeles and Principal Investigator for ASPIRO, Astellas Gene Therapies, Regina Espanol Suner, Scientist II, Vector Biosciences, Astellas Gene Therapies, Isabelle Richard, Chief Scientific Officer, Atamyo Therapeutics, Roger Hajjar, Therapeutic Sector Leader, Cardiac Gene Therapy, AskBio, Serge Braun, Head of Neuromuscular Strategy, Genethon, Sharif Tabebordbar, Research Scientist, Broad Institute of MIT and Harvard, Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association, Stan Froehner, Co-Founder and Chief Executive Officer, Myosana Therapeutics, Suryanarayan Somanathan, Head of AAV Gene Therapy, Rare Disease Research Unit, Pfizer, Tudor Fulga, Vice President, Gene Editing, Vertex Cell and Genetic Therapies