The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…
The U.S. Food and Drug Administration (FDA) has pushed back by more than one year a decision on Roctavian, the potential first gene therapy for adults with hemophilia A. In a complete response letter that took the therapy’s developer, BioMarin by surprise, the FDA said it needed…
A single dose of the investigational gene therapy BAY 2599023 safely promotes a sustained production of factor VIII (FVIII),  effectively preventing spontaneous bleeds in people with severe hemophilia A, a Phase 1/2 trial shows. Two of the six patients treated to date in this clinical study, both given the…
Treatment with Eloctate (efmoroctocog alfa) was well-tolerated and effective at preventing and treating bleeding episodes in children with severe hemophilia A who had never received any form of therapy for their disease, a Phase 3 trial shows. The study had enrolled boys with severe hemophilia A younger than…
A single infusion of valoctocogene roxaparvovec, BioMarin Pharmaceutical’s investigational gene therapy, can lead to the sustained production of factor VIII (FVIII) in people with hemophilia A, according to a biopsy study that showed the presence of the treatment’s gene constructs in the liver over nearly four…
A single dose of the investigational gene therapy FLT180a can lead to clinically meaningful, sustained increases in the activity of factor IX (FIX), effectively preventing bleeds and the need for replacement therapies in people with severe hemophilia B, a Phase 1/2 clinical trial shows. These results support the…
Note: This story was updated July 20, 2020, to note that Hemlibra binds to activated FIX and FX, rather than to FIX and FX. When given as preventive treatment to avoid spontaneous bleeds in people with hemophilia A with inhibitors, Hemlibra (emicizumab) continues to show a…
A single dose of the investigational gene therapy SPK-8011 is safe and leads to durable production of factor VIII (FVIII) for up to three years, effectively lowering the frequency of spontaneous bleeds in men with hemophilia A, preliminary data from a Phase 1/2 trial show. The findings were…
Freeline has raised $120 million to support the launch of a pivotal clinical trial of FLT180a, the company’s lead gene therapy candidate for the treatment of hemophilia B. FLT180a uses AAVS3, Freeline’s proprietary adeno-associated virus (AAV) protein shell, or capsid, to infect human liver…
CSL Behring has acquired exclusive global rights to commercialize AMT-061, a potential gene therapy for hemophilia B now in a pivotal clinical trial, from its developer uniQure. Under terms of the agreement, uniQure will receive an upfront payment of $450 million from CSL Behring, and is eligible…
Get regular updates to your inbox.