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Gene Therapy for Rare Disorders 2022

Gene Therapy for Rare Disorders 2022


Date of beginning

Monday, 07 March 2022


4 days




United States


Olivia Bull


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Gene Therapy for Rare Disorders is dedicated to overcoming the unique drug development challenges encountered in transforming gene therapy concepts into reality for rare disease patients in need. As the gene therapy field strives to strike the balance between clinical efficacy and product safety in the context of mounting regulatory scrutiny, Gene Therapy for Rare Disorders 2022 will provide in-depth insights from experts who know how to drive clinical progress, optimize manufacturing, and demonstrate the value of gene therapy products to regulators, payers, and patients. Across four days, this meeting will unite 600+ leading experts in Boston from innovative biotechs, large pharma, academia, and key service provider companies, delivering actionable insights and valuable networking opportunities to accelerate gene therapy progress. This is your comprehensive guide to define your commercial path forward. Tickets Prices:Drug Developer - FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day USD 5495.00,Drug Developer - 2 Day Conference + Workshop Day USD 4297.00,Drug Developer - 2 Day Conference + Discussion Day USD 4297.00,Drug Developer - 2 Day Conference USD 3099.00,Standard - FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day USD 6495.00,Standard - 2 Day Conference + Workshop Day USD 5297.00,Standard - 2 Day Conference + Discussion Day USD 5297.00,Standard - 2 Day Conference USD 4099.00 Speakers: Cristina Baricordi, Senior Scientist, AVROBIO, Deborah Ascheim, CMO, StrideBio, Eduard Ayuso, CTO, DiNAQOR, Luigi Barbarossa, Senior Research Associate, AVROBIO, Barbara Bonamassa, Quality Assessor, AIFA , Mike Byrne Senior Director, Analytical Development & Product Quality, REGENXBIO, Ruth Castellanos, Associate Director, Stridebio, Ying Cai, Senior Director, Process Development, Ultragenyx, Daniel Chung, CMO, SparingVision, Fernanda Copeland, Executive Director, Global Head of Patient Advocacy & Engagement, AVROBIO, Jed Chatterton, VP, Gene Therapy, Gemini Therapeutics, Kent Christopherson, Senior Director, Medical Affairs, Orchard Therapeutics, Eric Faulkner, VP, Global RWE, Novartis Gene Therapies, Rebecca Derwin, Director, Early Commercial Strategy, Rare Programs, REGENXBIO, Scott Dorfman, CEO, Odylia Therapeutics, Simon Delagrave, SVP, Ring Therapeutics, David Favre, CSO, InnoSkel, Jennifer Helfer, Senior Director, Patient Advocacy & Engagement, Encoded Therapeutics, Rajiv Gangurde, CTO, SparingVision, Xiaotong Fu, Senior Engineer, Biogen, Dawn Henke, Senior Scientific Program Manager, Standards Coordinating Body (SCB), Jingjing Jiang, Associate Director, Gene Therapy, Poseida Therapeutics, John Jefferies, Professor and Chief, Division of Adult Cardiovascular Diseases, University of Tennessee Health Science Center, Vibha Jawa, Executive Director, Bristol Myers Squibb, Angela Johnson, Senior Director, Regulatory Strategy, Roivant, Eric Kelsic, CEO, Dyno Therapeutics, Kei Kishimoto, CSO, Selecta Biosciences, Klaudia Kuranda, Head of Immunology, Spark Therapeutics, Genevieve Laforet, VP, Clinical Development, BridgeBio Gene Therapy, Janet Lambert, CEO, ARM, Michael Ladd, VP, Regulatory Science, Orchard Therapeutics, Maryanne Maliwat, Director, Pricing, Reimbursement & Market Access, REGENXBIO, Nick Li, Senior Director, Global/US Market Access Lead, uniQure, Peter Marks, Director, CBER, FDA, Séverine Marconi, EU Senior Director, Regulatory Affairs, AskBio, Anju Mahesh, Senior Director, Regulatory CMC, Ultragenyx, Chris Mason, CSO, AVROBIO, Leslie Meltzer, CMO, Orchard Therapeutics, Sean McAuliffe, Chief Commercial Officer, Taysha Gene Therapies, Amy Nayar, VP, US Patient Advocacy & Government Affairs, Novartis Gene Therapies, Emily McGinnis, Chief Patient Officer & Head of Government Affairs, Taysha Gene Therapies, Federico Mingozzi, CSO, Spark Therapeutics, Manish Muhuri, Instructor, University of Massachusetts Medical School, Asif Paker, VP, Clinical Development, SwanBio Therapeutics, Christopher Park, Principal Analyst and Data Analytics Advisor, Medicaid & CHIP Payment & Access Commission (MACPAC), Clark Paramore, Head of Value Demonstration, VP HEOR, bluebird bio, Palani Palaniappan, CTO, Aruvant, Kim Raineri, Chief Manufacturing & Technology Officer, AVROBIO, Markus Peters, CEO, Aevitas Therapeutics, Natalie Reash, Research Physical Therapist,Nationwide Children’s Hospital, Tayler Renshaw, Associate Director, Regulatory Affairs CMC, Astellas Gene Therapies, Abraham Scaria, CSO, IVERIC Bio, Jessica Riviere, VP, Global Patient Advocacy & Patient Engagement, Ultragenyx, Michael Sherman, EVP & CMO, Point32 Health, Sabah Sallah, SVP, Freeline Therapeutics, Lisa Stanek, VP, Translational Sciences, Affinia Therapeutics, Magali Taiel, CMO, GenSight Biologics, Mauricio Umana, Associate Director, Global Regulatory CMC Lead, Biogen, Shaun Vigeant, Senior Director, Talent Acquisition & HRBP, Intellia Therapeutics, Alexandra Watt, Module Manager, Advanced Therapies, Beacon Targeted Therapies, Courtney Wells, VP, Clinical Operations & Clinical Affairs, Jaguar Gene Therapy, James Warren, VP, Pharmaceutical Development, Ultragenyx, Pierre-Axel Vinot, Associate Director, CMC Portfolio Management, SparingVision, Eric Yearley, Associate Director, Analytical Development, BridgeBio Gene Therapy, Faraz Ali, CEO, Tenaya Therapeutics, Genine Winslow, CEO, Chameleon Biosciences, Karl Whitney, VP, Regulatory Affairs, Precision Biosciences, Cathy Garabedian, Senior Director, New Product Planning, Keros Therapeutics, Isabella Palazzolo, Associate Director, Regulatory CMC, Gene Therapy, Biogen, Lucas de Breed, Managing Director, August Care, Mariana Nacht, CSO, LogicBio, Kaye Spratt, Chief Regulatory Officer, BridgeBio Gene Therapy, Mo Heidaran, Ph.D., Vice President – Technical, Regulatory & Access, Parexel, Brenda Faiola, Ph.D., DABT Director Nonclinical Development, Rho, Joseph Watson, Ph.D., Associate Director Regulatory Strategy, Rho, Kelsey Behrens, Ph.D., Research Scientist, Rho Date and Time: Starts: Mon, Mar 7, 2022 ( 7:50 AM) and Ends: Thu, Mar 10, 2022 ( 5:00 PM) Venue details: Hynes Convention Center, 900 Boylston Street, Boston, Massachusetts, 02115, United States