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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…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

CB 2679d-GT, an investigational gene therapy for hemophilia B being developed by Catalyst Biosciences, can increase factor IX (FIX) levels and significantly reduce bleeding, early studies show. The data, “Combination of a Novel Chimeric AAV Capsid and Potency Enhanced FIX Variant for Hemophilia B…

The European Medicines Agency (EMA) has approved 21-day dosing intervals for CSL Behring‘s Idelvion (rIX‐FP) as a prophylactic treatment for adults with hemophilia B.  Idelvion — which is…

People with hemophilia A in the U.K. will soon have greater access to Elocta (efmoroctocog alfa) under a two-year agreement between Sobi and the National Health Service (NHS), the company announced. Elocta was approved by the European Commission for the treatment and prevention of bleeding in all…

Sobi and Sanofi announced an additional donation of up to 500 million international units (IUs) of clotting factor therapy in support of the World Federation of Hemophilia‘s Humanitarian Aid Program. The clotting factor goes toward treating people with hemophilia in developing countries where access to medicine is…