6th Gene Therapy for Rare Disorders 2023 Sheraton Boston Hotel, Boston

2022 has been a landmark year for the global gene therapy space. With August came the green light for bluebird bio’s Zynteglo in the US, the first FDA approval for a gene therapy in over three years. Hot off its heels we saw the accelerated approval of another bluebird bio product, Skysona. Overseas, July and August brought EMA approvals for PTC Therapeutics’ Upstaza and BioMarin’s Roctavian, the first gene therapies for AADC deficiency and Haemophilia A respectively.

As the gene therapy field continues to break records, there are still significant challenges to overcome relating to safety, efficacy, and accessibility. The 6th Annual Gene Therapy for Rare Disorders 2023 Summit remains devoted to showcasing the top case studies and strategic learnings from the past year. With an expert speaking faculty devoted to bringing safer and more effective gene therapies to rare disease patients, key questions will be answered on how best the field can overcome regulatory, clinical, manufacturing and pricing bottlenecks to progress gene therapies into and through the clinic.

500+ leading experts from innovative biotechs, large pharma, academia and key service providers will be reuniting in Boston for 2023 to capitalize on recent success stories and collaborate over the most pressing industry challenges. An event vital to unlocking the full potential of your rare gene therapy program, join us to keep your finger on the pulse and set up for success in 2023.

Speakers: Faraz Ali, Chief Executive Officer, Tenaya Therapeutics, Deborah Ascheim, Chief Medical Officer, Stridebio, Eduard Ayuso, Chief Executive and Technology Officer, DiNAQOR, Sudha Babu, Regulatory CMC Leader, Spark Therapeutics, Joe Balleydier, Vice President Process Development, Jaguar Gene Therapy, Mattia Calissano, Head of Drug Safety and Risk Management, Orchard Therapeutics, Mindy Cameron, Patient Advocacy Consultant, AdvocacyWorks, Lisa Carlton, Vice President, Regulatory Affairs, REGENXBIO, Vivian Choi, Head of Global Gene Therapy Research, Takeda, Angela Columbano, Head of Business Development and Partnership, Genethon, Richard Colvin, Chief Medical Officer, bluebird bio, Jean Philippe Combal, Chief Executive Officer and Co-Founder, Vivet Therapeutics, Eric Crombez, Chief Medical Officer, Ultragenyx, Melody Dai, Senior Director Regulatory Affairs, CMC Adverum, Doug Danison, Head of Commercial Strategy and Operations, CGT Bayer, Kumar Dhanaeskharan, Senior Vice President, Technical Operations, Renovacor, David Dobnik, Senior Research Associate, Slovenian National Institute of Biology, Debbie Drane, Senior Vice President, Global Commercial Development, CSL Behring, Carrie Edwards Wolkoff, Gene Therapy National Account Management/Commercial Director Gov, and Federal US, Michael Grosso Vice President, Global Head of Clinical Development, Specialty Medicine Daiichi Sankyo, Marianne Hamilton Lopez, Senior Research Director of Biomedical Innovation, Duke-Margolis Center for Health Policy, Harm Hermsen, Vice President, Global Regulatory Affairs, uniQure, Katherine High President, Therapeutics AskBio, Kaha Hizanishvili Former Chief, Provider and Pharmacy Programs MassHealth, Elizabeth Hook Senior Director, Regulatory Affairs, Homology Medicines, Timothy Hunt Chief Executive Officer, Alliance for Regenerative Medicine, Rita Johnson-Greene Vice President, Field Promotion and QTC Engagement, bluebird bio, John Johnston, Clinical Assessor, Medicines and Healthcare Products Regulatory Agency (MHRA), Lawrence Kau, Global Commercial Development Lead, Rare Disease Gene Therapy, Spark Therapeutics, Niamh Kinsella Director, Global Regulatory CMC Early Development Gene Therapy Lead, Biogen, Kathleen Kirby, Principal, Viridian Strategies, Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences, Klaudia Kuranda, Head of Immunocolgy, Spark Therapeutics, Ying Liu Associate Director, Cell Culture Process Development, Sanofi, Geoff Mackay President and Chief Executive Officer AVROBIO, Peter Marks Director, CBER US Food and Drug Administration (FDA), Tristan Marshall Vice President, Process Development REGENXBIO, Craig Martin Chief Executive Officer Global Genes, Yoshiaki Maruyama Review Director, Office of Cellular and Tissue-Based Products Pharmaceuticals and Medical Devices Agency (PMDA), Brad Mickey Director, Manufacturing, MSAT and Engineering Astellas, Giridhar Murlidharan Director, Translational Science Affinia Therapeutics, Mariana Nacht Chief Scientific Officer Logicbio, Daniel O’Connor Deputy Director, Innovation Accelerator and Regulatory Science Medicines and Healthcare Products Regulatory Agency (MHRA), Christina Ohnsman Senior Lead, Clinical Development REGENXBIO, Samantha Parker Chief Patient Access Officer InnoSkel, Nicole Paulk Assistant Professor University of California, San Francisco, John Petrich Director of Pharmacy, Residency Program Director, Investigational Drugs and Research Cleveland Clinic, Sarah Pitluck Former Vice President and Head Of Global Pricing and Reimbursement Strategy Spark Therapeutics, Lynne Quittell Associate Director, Data and Safety Monitoring Board Cystic Fibrosis Foundation, Jessica Riviere Vice President, Patient Advocacy and Patient Engagement Ultragenyx, Oscar Segurado Chief Medical Officer ASC Therapeutics, Albert Seymour Chief Medical Officer Homology Medicines, Courtney Silverthorn Associate Vice President, Research Partnerships FNIH, Rakesh Sindhi Professor of Surgery University of Pittsburgh, Nripen Singh Head, Process and Product Development Passage Bio, Gregory Stewart Executive Consultant Alcyone Life Sciences, Kevin Strauss Medical Director UMass Medical School, Sitra Tauscher-Wisniewski Vice President, Clinical Research Novartis, Kris Thiruvillakkat Senior Director – Global Evidence Market Access and Pricing CSL Behring, Cory Thomas Lead Engineer Sarepta Therapeutics, Mark Trusheim Strategic Director, NEWDIGS and Visiting Scientist MIT, Farrah Vogel-Javeri Researcher Beacon, Jim Wang Chief Regulatory Officer Adverum, Chris Williams Principal Engineer Roche, Ashley Winslow President and Chief Scientific Officer Odylia Therapeutics, Genine Winslow Chief Executive Officer and Founder Chameleon Biosciences, Weicheng Wu Vice President, Global Regulatory Affairs PTC Therapeutics, Vivienne Xiong Senior Scientist, Analytical Development REGENXBIO, Frank Zhang Head of Commercial uniQure, Julie Jordan Senior Vice President, Head of Clinical Development and Operations Homology Medicines, Martine Zimmermann Senior Vice President, Head of Regulatory and QA Alexion, Omer Munir Senior Director uniQure, Bert Yao Vice President Clinical Development PTC Therapeutics, Emma James Vice President, Medical and Patient Affairs Encoded Therapeutics, Federico Mingozzi Chief Science and Technology Officer Spark Therapeutics