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Gene Therapy for Muscular Disorders

Gene Therapy for Muscular Disorders


Date of beginning

Tuesday, 25 May 2021


3 days




Ellie Saunders


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With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. Despite the successes, there are still withstanding challenges for gene therapy developers targeting the muscle when it comes to optimizing delivery and navigating toxicity to develop truly safe and transformative therapeutics for muscular disorders patients. The first industry-dedicated Gene Therapy for Muscular Disorders Summit will focus on overcoming the translational and clinical challenges of developing genetic therapies targeting muscular disorders, including Duchenne Muscular Dystrophy (DMD), Limb-Girdle Muscular Dystrophy (LGMD), Pompe Disease and X-Linked Myotubular Myopathy. Join your fellow industry leaders from the likes of Sarepta Therapeutics, PTC Therapeutics, Solid Biosciences, Pfizer, AskBio and Novartis at this digital event in March. With a focus on AAV and gene editing technologies, this niche 3-day agenda has been specifically designed to help industry experts to: Optimize your promoter choice and navigate IP challenges for more efficient delivery to muscular targetsDiscover the next generation capsids being developed for improved muscle selectivity and reduced immunogenic responsesUnderstand the biology of different immunogenic responses and hear feedback from trialled strategies to ensure optimal patient safetyHear the latest research in muscular disorder biomarkersDefine clinically meaningful muscular endpoints to successfully and objectively measure muscular disease improvementTackle regulatory challenges to demonstrate gene therapy durabilityOvercome clinical challenges to manage patient heterogeneity for patient centric and meaningful trials Join your peers to share, learn and discuss at the first industry-focused Gene Therapy for Muscular Disorders. Overcome your immunogenicity challenges and mitigate toxicity with high doses targeting the muscle to successfully demonstrate durability with your gene therapy product. URLs:Brochure: Tickets:  Date and Time: On Tuesday May 25, 2021 at 8:00 am (ends Thursday May 27, 2021 at 5:00 pm) Prices:Industry Pricing - Conference + 3 Workshops (First Release): USD 2096.00,Industry Pricing - Conference + 2 Workshops (First Release): USD 1897.00,Industry Pricing - Conference + 1 Workshop (First Release): USD 1698.00,Industry Pricing - Conference Only (First Release): USD 1499.00,Academic Pricing - Conference + 3 Workshops (First Release): USD 2696.00,Academic Pricing - Conference + 2 Workshops (First Release): USD 2397.00,Academic Pricing - Conference + 1 Workshop (First Release): USD 1298.00,Academic Pricing - Conference Only (First Release): USD 1199.00 Speakers: Michael O'Callaghan, Senior Vice President, Translational Medicine, AskBio, Michael Roberts, Chief Technology Officer, AskBio Europe, Pat Furlong, Founding President and Chief Executive Officer, Parent Project Muscular Dystrophy, Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association, Stan Froehner, Co-Founder, Myosana Therapeutics, Takashi Kishimoto, Chief Scientific Officer, Selecta Biosciences, Lee Sweeney, Professor and Director, Myology Institute, University of Florida, Luk Vandenberghe, Grousbeck Associate Professor in Gene Therapy, Harvard; Co-Founder and Chief Science Advisor, Affinia Therapeutics, Mark Milton, Executive Director, PK Sciences, Novartis, Marla Weetall, Vice President, Pharmacology, PTC Therapeutics, Mathew Pletcher, Senior Vice President, Head of Research, Audentes Therapeutics, Michael Binks, Vice President and Head of Rare Disease Clinical Research, RDRU, Worldwide Research and Development, Pfizer, Carl Morris, Chief Scientific Officer, Solid Bio, Dongsheng Duan, Professor, University of Missouri School of Medicine, Jeffrey Chamberlain, Professor, Departments of Neurology, Medicine, and Biochemistry, University of Washington, John Kamerud, Director, Bioanalytics, Pfizer, Kevin Flanigan, Director, Center for Gene Therapy, Nationwide Children's Hospital; Professor, The Ohio State University, Kevin Whittlesey, Vice President, Translational Medicine, Project Leader for Neuromuscular Disorders, 4DMT, Alison McVie-Wylie, Vice President, Disease Area Executive, DMD, Vertex Cell and Genetic Therapies, Anne-Ulrike Trendelenburg, Director, Head of MSD Cambridge, Novartis Institutes for Biomedical Research (NIBR), Musculoskeletal Disease Area (MSD), Novartis, Barry Byrne, Associate Chair of Pediatrics and Director, Powell Gene Therapy Center, University of Florida