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New Gene Therapy For Hemophilia Shows Potential As Safe Treatment

March 12, 2015

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 Forms Partnership to Focus on Gene Therapy for Hemophilia

January 30, 2015

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 Establishes Gene Therapy Partnership

December 8, 2014

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 […]

Promising Gene Therapy Results in Treating Hemophilia B

November 20, 2014

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 […]

Stanford University: New Form of Gene Therapy Could Cure Hemophilia in Mice

October 30, 2014

[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 […]

Bayer to Develop Hemophilia Gene Therapy Treatment

June 23, 2014

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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