Andrea Lobo, PhD, science writer —

Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing. The move is backed by preclinical studies in nonhuman primates that show the treatment led to…

Centessa Pharmaceuticals is discontinuing the clinical development of SerpinPC, its experimental under-the-skin therapy for people with hemophilia A and hemophilia B with or without inhibitors. The therapy was being evaluated in several Phase 2 clinical trials. The decision follows the recent approval of Pfizer’s Hympavzi…

People with hemophilia are more likely to be infected with the SEN virus (SENV), which is associated with blood transfusion hepatitis, or liver inflammation, than healthy people, a study in Iran suggests. The virus was detected in more than half of patients with hemophilia A and most commonly in…

The Spanish Interministerial Commission on the Pricing of Medicines, known as CIPM, has issued a positive recommendation for national reimbursement of Hemgenix (etranacogene dezaparvovec) for eligible people with hemophilia B, according to CSL Behring, the company that markets the gene therapy. The recommendation means that patients in Spain should…

Note: This story was updated Oct. 15, 2024, to note results from the subset of patients with inhibitors participating in BASIS are now expected next year. A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for…

Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy used in treatment, for people with hemophilia, a new study from China found. The study did reveal, however, that, compared with adults, children with hemophilia had a significantly lower…

MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…

Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…

A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix (fidanacogene elaparvovec), developed by Pfizer, to treat adults with severe and moderately severe hemophilia B. The European Commission will review the recommendation from the EMA’s Committee for Medicinal…