Adults and children with severe hemophilia A who are receiving Nuwiq (recombinant human coagulation factor VIII) as their first replacement therapy have a similar risk of developing inhibitors as those treated with plasma-derived therapies containing von Willebrand factor, the final results of the NuProtect trial show.
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ASC Therapeutics is partnering with Vigene Biosciences to support the manufacturing process of its current and future gene therapy clinical platforms — including its ongoing hemophilia A program. “We are proud to welcome Vigene, a global leader in gene therapy process development and GMP [Good Manufacturing…
Generation Bio said it received $110 million in funding to bring its innovative, non-viral gene therapy for lead programs hemophilia A and phenylketonuria into investigational new drug-enabling clinical trials. Gene therapy is an investigational treatment technique that uses genes or genetic material to treat or prevent…
BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec safely and significantly reduced bleeding events — including in target joints — and the use of prophylactic clotting factor VIII in adults with severe hemophilia A, data from a Phase 1/2 study show. These results, along with an interim analysis of…
Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies. The findings, presented at the 61st American Society of Hematology (ASH) Annual Meeting…
Bayer’s investigational gene therapy BAY 2599023 safely and effectively increased the levels of clotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated in a Phase 1/2 clinical trial, preliminary data show. The ongoing trial (NCT03588299; 2017-000806-39) is enrolling…
A programmable mini-pump able to provide regular doses after surgery of FVIIa to hemophilia A patients with inhibitors — those resistant to standard FVIII replacement therapy — successfully maintained expected levels of blood clotting factors, a small study reports. Attending surgical nurses also voiced satisfaction with the pump’s use, reporting a…
A lower dose of regular, preventive therapy with factor VIII concentrates was better at reducing bleeding events and improving joint function than on-demand treatment in children with hemophilia in Thailand, a study shows. The study, “Prophylactic vs episodic treatment to prevent bleeds and preserve joint function…
A modified version of the coagulation protein factor X was able to significantly reduce bleeding in a mouse model of hemophilia A, and may offer a way of treating people with this disease due to factor VIII deficiency, a study suggests. Lab work also showed a potential for treating people…
Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up…