An investigational factor VIIa (FVIIa) therapy called GEN-0828 may be able to minimize blood loss and accelerate blood clotting at lower doses than NovoSeven, an approved medication used to control bleeding in hemophilia patients, according to a study in a mouse model of hemophilia B. Still, both treatments…

The World Federation of Hemophilia (WFH) has launched a registry to monitor long-term outcomes of safety and effectiveness for people with hemophilia who receive gene therapy treatment. The aim of the WFH Gene Therapy Registry (GTR) is to collect data for all patients who have gene therapy, whether through…

Treatment with the experimental gene therapy fidanacogene elaparvovec led to significant reductions in bleeding rates for men with moderately severe to severe hemophilia B who participated in a Phase 3 clinical trial. That’s according to top-line data from the study BENEGENE-2 (NCT03861273), recently announced by the therapy’s developer,…

One-time treatment with the investigational gene therapy SPK-8011 led to sustained low bleed rates for people with hemophilia A in a Phase 1/2 clinical trial. That’s according to data of up to 5 years of follow-up presented by the therapy’s developer, Spark Therapeutics, at the annual meeting of…

A committee of the European Medicines Agency (EMA) has issued a positive opinion recommending the one-time gene therapy etranacogene dezaparvovec be granted conditional marketing authorization to treat appropriate adults with hemophilia B. The recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will now be…

The positive safety and efficacy profile of Hemgenix (etranacogene dezaparvovec) — the first and only approved gene therapy for adults with hemophilia B — was sustained for two years, according to data from a Phase 3 trial, CSL Behring has announced. Findings were generally similar to previous…

A review is continuing into a resubmitted application for approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for adults with severe hemophilia A, with a planned advisory committee meeting having been canceled. The U.S. Food and Drug Administration (FDA) had requested, but now no longer intends, to meet with…

The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…

The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…

Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…