BioMarin Investigational Gene Therapy Updates

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An update for the hemophilia community from BioMarin regarding a previously reported serious adverse event in the ongoing hemophilia A gene therapy [valoctocogene roxaparvovec, BMN 270] Phase 3 study that was deemed, by the Independent Data Safety Monitoring Committee and the company, very unlikely to be related to the investigational therapy. Clinical Development Program Update […]

FDA Accepts CSL Behring’s Biologics License Application for Etranacogene Dezaparvovec for Priority Review  

Global biotherapeutics leader CSL Behring, a business of CSL, announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA), for priority review, for etranacogene dezaparvovec (also known as CSL222), an investigational gene therapy for the treatment of adults with hemophilia B. Etranacogene dezaparvovec was specifically designed to make near-normal […]

Pfizer Reports First Quarter 2022 Update of Gene Therapy Trial

Read the original press release. During a recent quarterly stakeholder report, Pfizer announced that in March 2022 the U.S. Food and Drug Administration lifted a clinical hold that had been placed on the Phase 3 AFFINE study in November 2021 following the observance of Factor VIII levels greater than 150% in some study participants. Pfizer […]

uniQure and CSL Behring Achieve Primary Endpoint in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B

Press release source: uniQure and CSL Behring via Globe Newswire CSL Behring, a global biotherapeutics leader, and uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that etranacogene dezaparvovec, an investigational adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately […]

Astellas Reports Fourth Death In Orphan Disease Gene Therapy Clinical Trial

The following is a news release from Astellas Pharma Inc. Read the release. Astellas Pharma Inc. reports that the ASPIRO clinical trial participant, who developed a serious adverse event as described on September 1, passed away on September 9. The cause of death is still pending. Astellas is gathering the relevant clinical information and incorporating […]

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

Read the full press release from CSL Behring here. Global biotherapeutics leader CSL Behring announced the closing of its global Commercialization and License agreement with uniQure for etranacogene dezaparvovec, a novel gene therapy for the treatment of hemophilia B. Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown  to result in near-normal […]

New Gene Therapy Treatment Proves Effective In Dogs

Note: The following is an edited form of a press release from the University of North Carolina School of Medicine. The original release can be read here. A single injection. That’s all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration […]