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.