News
FDA Approves First Gene Therapy to Treat Adults with Hemophilia B
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
On November 22, the U.S. Food and Drug Administration approved Hemgenix (etranacogene dezaparvovec), an adeno-associated virus (AAV) vector-based gene therapy. It is…
Unanswered Questions Remain in Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
A recent commentary published in the journal Haemophilia, posits that while clinical trials for hemophilia gene therapy approach conclusion, there remain significant unanswered…
FDA Recommends Additional Data for BioMarin’s Investigational Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Biomarin announced that they received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) on August 18th for their…
CSL Acquires Hemophilia B Gene Therapy Candidate from uniQure
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
CSL Behring recently announced that it has agreed to acquire exclusive global license rights from uniQure, to commercialize etranacogene dezaparvovec (AMT-061), an…
Two Gene Therapy Medscape Activities Recertified
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
The National Hemophilia Foundation (NHF) is pleased to announce that two educational activities in the “Clinical Advances in Gene Therapy for Hemophilia”…
Casebia Presents Dual Approach to Gene Therapy
Gene Therapy, Industry News & Research, Living with a Bleeding Disorder
Cambridge, Massachusetts-based Casebia Therapeutics recently presented new research on a novel therapy being developed for patients with hemophilia A. Casebia Therapeutics is…