Factor VIII Gene Therapy for Hemophilia A

In a phase I clinical trial, nonviral delivery of Factor VIII using the genetically modified cells of homophilia A patients demonstrated that four of the first six patients showed decreased bleeding, with two patients having no spontaneous bleeds for close to a year following treatment. The results were presented at the annual meeting of the American Society of Hematology. The trial was conducted by researchers from Beth Israel Deaconess Medical Center (NY, NY, USA) and Harvard Medical School (Boston, MA, USA).

In the trial, a small sample of a patient's cells were removed in an outpatient procedure and sent to a manufacturing facility where the cells were genetically engineered to produce the blood-clotting protein Factor VIII for extended periods of time. The cells were then injected back into the patient, where they were shown to be safe and well tolerated. There were no serious adverse events. The procedure was developed by Transkaryotic Therapy (TKT, San Francisco, CA, USA). The company's technology is designed to take advantage of a patient's ability to synthesize therapeutic proteins for extended periods, potentially providing improved therapeutic outcomes, a reduction of side effects, improved patient compliance, and cost reductions.

"We are very excited by the clinical activity we have seen this early on in the program, but it is important to emphasize to the hemophilia community that we still have a great deal of work ahead to develop a safe and effective treatment,” said Richard F. Selden, M.D., president and CEO of TKT.



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