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…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively reduces bleeding rates in children younger than 12 with hemophilia type A, regardless of development of inhibitors (antibodies) against synthetic factor VIII (FVIII), data from two Phase 3 studies show. Hemlibra is a type of replacement therapy that mimics…

Novo Nordisk is inviting children and young adults with hemophilia A and B to take part in its observational study of how this disease affects brain development, thinking, and behavior. Results will help researchers better understand how advances in hemophilia treatment, like routine prophylaxis, impact the brain by establishing…