6th Gene Therapy for Rare Disorders 2023

Conference themes
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.

Scientific Society
Hanson Wade

Notes
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 and More

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Ms. Emily Birt