Our next panel, “The ABC’s of Gene Therapies, Starting with the Importance of AAV”, featured Matthew Klein, MD, MS, FACS, COO of PTC Therapeutics, Inc., and Matthew Kapusta, CEO of uniQure. The panel was moderated by Kristen Kluska.

• Matthew Klein discussed the data around Upstaza, an AAV gene therapy for AADC Deficiency that recently led to EU marketing authorization.

• Dr. Kapusta discussed uniQure’s rich history in gene therapy, which led to a previous approval, its partner submitting approvals for EtranaDez in hemophilia B, and the pipeline expansion and early evidence.

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