Hemophilia Federation of America is a national nonprofit organization that assists, educates, and advocates for the bleeding disorders community.
Research showed that bleeding events were drastically decreased in animals with Hemophilia B. Using a viral vector to swap out faulty genes proved safe and could be used for the more common Hemophilia A. _______________________________ A multi-year, ongoing study suggests that a new kind of gene therapy for Hemophilia B could be safe and effective […]
Biogen Idec, Fondazione Telethon, and Ospedale San Raffaele have entered into a worldwide collaboration to jointly develop gene therapies for the treatment of both hemophilia A and B. The agreement will combine San Raffaele – Telethon Institute for Gene Therapy’s (TIGET) extensive expertise in creating new gene therapy strategies and developing them from the bench to bedside […]
Pfizer Inc. announced on Monday, December 8, 2014 two strategic decisions to expand the company’s rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a program incorporating a bio-engineered AAV vector for the potential […]
On November 19, St. Jude Children’s Research Hospital announced the results of their ongoing gene therapy partnership with University College London and the Royal Free Hospital. The report indicates success in the trial, which used a modified adeno associated virus (AAV) 8 to deliver genetic material for making Factor IX in Hemophilia B patients. In […]
[glossary_exclude] A technique developed by Stanford researchers could provide a safer, longer-lasting method of replacing faulty, disease-causing genes with working copies. The ability to pop a working copy of a faulty gene into a patient’s genome is a tantalizing goal for many clinicians treating genetic diseases. Now, researchers at the Stanford University School of Medicine […]
Dimension Therapeutics, a Cambridge biotechnology startup, has struck a deal worth up to $252 million with the pharmaceutical giant Bayer HealthCare to develop a gene therapy to treat hemophilia, a rare disease that prevents blood from clotting. The goal of the prospective treatment, which is not yet in clinical trials, is to repair a faulty […]
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