News

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,…

Three years after receiving the one-time gene therapy Roctavian (valoctocogene roxaparvovec) at trial, the majority of men with severe hemophilia A in the Phase 3 GENEr8-1 study were off standard replacement therapies and did not experience any bleeds requiring treatment. That’s according to new data announced in a…

The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be used by clinical labs to detect a deficiency in factor IX (FIX), the cause of hemophilia B. The chromogenic, or color-based, test developed by Precision BioLogic is reported…

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…

Preventive treatment with Hemlibra (emicizumab) appears to be safe and helps keep bleeding under control in babies who have severe hemophilia A. The number of bleeds was “zeroed out” in nearly half of the babies, and most had zero bleeds requiring treatment. That’s according to interim results from…

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 European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors. “We’re very pleased that the CHMP’s recommendation brings us closer to potentially transforming the day-to-day lives of people in the…

SerpinPC, a potential treatment for all types of hemophilia, from the drug development pipeline at Centessa Therapeutics, was found to be safe and to reduce patients’ bleeding episodes for up to 1.5 years. In fact, the therapy candidate has the potential to nearly “zero out” the number of…

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…