Re-administering a viral vector-based gene therapy for hemophilia A was shown to be safe and to achieve long-term improvement in a dog model of the disease — even after neutralizing antibodies were evident following the initial treatment. The study, “An observational study from long-term AAV re-administration in…

Past infection with the hepatitis C virus (HCV) is the major risk factor for hepatocellular carcinoma (HCC), the most common type of liver cancer, a large study analyzing more than 18,000 U.S. inpatients with hemophilia reported.

The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the…

While there is no cure for hemophilia, it is very possible to live with this genetic bleeding disorder and maintain a good quality of life. Proper nutrition and weight management combined with a safe and effective exercise plan monitored by a medical professional can make…

Sernova will use AgeX Therapeutics’ UniverCyte gene technology to create a cell-based therapy that delivers therapeutic levels of factor VIII (FVIII) — the blood clotting protein deficient in people with…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…

CSL Behring announced that new data from studies of two approved treatments for hemophilia — Idelvion and Afstyla, both recombinant coagulation factors — will be presented at the XXXII International Congress of the World Federation of Hemophilia (WFH) 2016 next week. CSL will present six posters highlighting Idelvion, a long-acting recombinant albumin fusion…

Me: “Hello?” Medical assistant: “Hi, Shellye, the doctor wants to see you ASAP to discuss your recent test results. She’s concerned.” Me: “Oh no, that doesn’t sound good. Can you tell me what’s going on?” Medical assistant: “We just got your baseline bone density tests back and they show you…

Preventive treatment with Hympavzi (marstacimab-hncq) can effectively reduce bleeding rates in people with hemophilia A or B who have inhibitors, or antibodies against standard replacement therapies, according to top-line data from a Phase 3 clinical trial. Hympavzi’s developer Pfizer said it’s now planning to discuss these…