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Gene Therapy for Metabolic Disorders

Gene Therapy for Metabolic Disorders


Date of beginning

Tuesday, 14 September 2021


3 days




United States


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More companies and institutions than ever before are embarking on development of gene therapies in metabolic disease, and they are encountering unique challenges, from fine-tuning dosing to defining clinically relevant biomarkers. Gene Therapy for Metabolic Disorders will unite large pharma and innovative biotechs to collectively overcome translational drug development barriers to deliver on the transformative promise of gene therapy approaches for patients in need. Incorporating in-depth insights into the leading metabolic disease programs, this event will ensure that you can optimize dosing to achieve the required enzyme levels without over-expression, define the necessary parameters for transitioning to first-in-human trials, and overcome unique challenges encountered in multi-organ systemic gene therapy delivery for complex metabolic indications. Don’t get left behind. Join colleagues from Preclinical, Translational and Clinical departments of leading companies including AVROBIO, Freeline Therapeutics and LogicBio to optimize your gene therapy approach and revolutionize the treatment of metabolic diseases. Tickets Brochure  Prices:Industry Pricing - Conference + 3 Workshops USD 3296.00Industry Pricing - Conference + 2 Workshops USD 2897.00Industry Pricing - Conference + 1 Workshop USD 2498.00Industry Pricing - Conference Only USD 2099.00Academic Pricing - Conference + 3 Workshops USD 2796.00Academic Pricing - Conference + 2 Workshops USD 2497.00Academic Pricing - Conference + 1 Workshop USD 2198.00Academic Pricing - Conference Only USD 1899.00 Speakers: Jay Barth, CMO, LEXEO Therapeutics, Cristina Baricordi, Senior Scientist at AVROBIO, Melanie Blank, Medical Officer, Clinical Team Leader in OTAT at CBER, Emily Fields Associate Director, Patient Advocacy at bluebird bio, Peter de Haan, CSO & Co-Founder at Amarna Therapeutics, John Jefferies, Director, Methodist Cardiovascular Institute at UTHSC, Kei Kishimoto, CSO at Selecta Biosciences, Mariana Nacht, CSO at LogicBio, Asif Paker, VP, Clinical Development at Swan Bio Therapeutics, Giuseppe Ronzitti, INSERM Researcher, Head of Immunology and Liver Gene Transfer at Laboratory Genethon, Rose Sheridan, VP, Translational Research at Freeline Therapeutics, Jakob Sieker, Medical Director, Gene Therapy at bluebird bio, Michele Stone, VP, Translational Development at Kriya Therapeutics, Gerard Vockley, Chief of Medical Genetics & Director of the Centre for Rare Disease Therapy at University of Pittsburgh, Sara Castegini, Senior Manager, Global Policy at bluebird bio, Stephanie Cherqui, Associate Professor at UCSD, Jingjing Jiang, Associate Director at Poseida Therapeutics, Cara O’Neill, Co-Founder and CSO Cure at SanFilippo Foundation Time: On Tuesday September 14, 2021 at 8:50 am (ends Thursday September 16, 2021 at 5:00 pm)