The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…

Researchers, working with mice, have developed a new way of producing substantial numbers of gene-corrected liver cells with the possibility of repopulation in vivo. The strategy, which relies on the liver’s unusual regenerative skills, may hold promise for the treatment of diseases such as hemophilia B. The study by scientists at Oregon Health & Science University,…

Prophylactic hemophilia treatment can save patients with severe forms of the disease from the long-term consequences of joint damage and other complications. A report by Dr. Suchitra S. Acharya, MD, of Hofstra Northwell School of Medicine in New York, gives a detailed overview of the current state and future perspectives…

https://www.youtube.com/watch?v=IhVOXnB-0KQ In this video from Bleeding Disorders Community, Ryan shares his hemophilia story. He was diagnosed as a baby with severe hemophilia but he doesn’t let the disease stand in the way of his passion for fitness. MORE: Improvement in gene therapy carriers key step in hemophilia…

The investigational gene therapy SB-525 is tolerated well by patients, and dose-dependent responses are positive, according to preliminary results of a Phase 1/2 clinical trial of hemophilia A. The potential gene therapy is being developed as a partnership between Sangamo Therapeutics and Pfizer. It consists of…

A multi-institute research collaboration has received a $12 million grant from the National Heart Lung and Blood Institute to understand what’s behind limitations in gene therapies currently being tested in people with hemophilia A. The goal is to use this information to develop safer and long-lasting gene therapies…

People with mild to moderate hemophilia, in particular women with the disease, face unique challenges, many that are specifically linked to a lack of information and insufficient engagement with other patients and healthcare providers, according to a panel of U.S. experts. In a recent study, these experts proposed new…

Sangamo Therapeutics and Pfizer have entered a global collaboration and license agreement to develop and market gene therapy programs for hemophilia A, including SB-525. SB-525 was cleared by the U.S. Food and Drug Administration (FDA) in January as an investigational new drug (IND) for the…

Mild to moderate hemophilia B affects the relationships of adult patients, children with the disease and their caregivers in aspects such as telling others about the diagnosis, selecting a partner, and feeling bullied, according to an American study. The research, “Mild-severe hemophilia B impacts relationships of…