Gene Therapy Latest News | Experimental Gene Therapy Studies

BioMarin Announces Stable and Durable Annualized Bleed Control for ROCTAVIAN™️ in Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A

08 January 2023

BioMarin Says FDA No Longer Plans Advisory Meeting for Roctavian

24 November 2022

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate

29 December 2022

FDA Approves First Gene Therapy to Treat Adults with Hemophilia B

23 November 2022

First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ Approved by European Commission (EC)
24 August 2022
BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
24 June 2022
Freeline Initiates Dosing of Second Cohort in B-LIEVE Dose Confirmation Trial of FLT180a in Hemophilia B
14 June 2022
Seroprevalence to adeno-associated virus type 6 in people with hemophilia B from a UK adult cohort
03 June 2022

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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