Conference Description:

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.

URL:
Tickets: https://go.evvnt.com/1439816-2?pid=154
Prices:
Drug Developer - FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day: USD 5595.00,
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 2999.00,
Standard - FULL ACCESS PASS: 2 Day Conference + Workshop Day AND Discussion Day: USD 7095.00,
Standard - 2 Day Conference + Workshop Day: USD 5597.00,
Standard - 2 Day Conference + Discussion Day: USD 5597.00,
Standard - 2 Day Conference: USD 4099.00

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 and More

Category: Conferences | Science, Health and Medicine | Pharmaceuticals

Date and Time: 20th March 2023 at 8:00 am to 23rd March 2023 at 5:00 pm