News

Regeneron Pharmaceuticals and Intellia Therapeutics are expanding their collaboration to develop hemophilia A and B treatments using the CRISPR/Cas9 gene editing technology. The two companies signed a six-year agreement in 2016 to develop, license, and commercialize gene editing-based therapies. “We’re pleased to expand our work…

A new combination of gene and cell-based therapy durably delivered therapeutic levels of factor VIII (FVIII) — the blood clotting protein missing or defective in people with hemophilia A — and eased bleeding in a mouse model of the disease, a study from the HemAcure Consortium shows.

BioMarin Pharmaceuticals’ investigational gene therapy valoctocogene roxaparvovec continues to safely and effectively prevent bleeding episodes and the need for prophylactic clotting factor VIII in adults with severe hemophilia A, four-year data from a Phase 1/2 clinical trial show. The results were submitted for presentation at the World Federation…

A new type of gene therapy that uses RNA instead of DNA led to rapid and prolonged production of therapeutic levels of factor VIII (FVIII) in a mouse model of hemophilia A, a study reported. The study, “Treatment of…

Infection by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the coronavirus causing COVID-19, can induce the onset of acquired hemophilia A (AHA), a case report has found. As such, blood coagulation should be monitored closely in patients hospitalized for COVID-19, scientists said. The study, “…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of ET3, an investigational gene therapy for people with hemophilia A. The decision followed a review of the company’s investigational new drug application (IND) requesting clearance of ET3…

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…

Novo Nordisk has issued a notice warning healthcare professionals that the hemophilia B treatment Rebinyn (nonacog beta pegol, or N9-GP) can interfere with some laboratory tests that measure blood coagulation. Following reports that people with severe COVID-19 may develop a blood-clotting condition similar to…

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, Maximilian, has cystic fibrosis (CF), making him particularly susceptible to COVID-19 and the complications that can arise from it.