Breakthrough in Hemophilia Gene Therapy

Industry News
The following is an excerpt from an article in Medical News Bulletin. Read the full article here.


Researchers at Spark Therapeutics and Pfizer tested the safety and efficacy of a new hemophilia gene therapy vector for the treatment of hemophilia B.

The body’s ability to stop bleeding and repair damaged blood vessels depends heavily upon a variety of clot-forming enzymes. Deficiency in one such enzyme, coagulation factor IX, results in the development of hemophilia B – a disorder in which a patient may bruise easily and spontaneously bleed from the nose, joints, or along the urinary tract.
A recent article published in the New England Journal of Medicine, reports the results of an early hemophilia gene therapy trial. The trial examined the safety and efficacy of the SPK-9001 therapeutic vector; a neutralized virus engineered to insert a hyperactive form of the factor IX gene into a patient’s DNA. Ten men with hemophilia B, with factor IX levels 2 percent of normal or below, received an intravenous injection of the vector and were monitored for up to 18 months afterwards. All participants were receiving factor IX transfusions prior to the start of the trial – seven preventatively and three as needed.
By the end of the trial the patients were producing, on average, 33.7 percent of the normal level of factor IX. After the gene therapy, bleeding decreased from an average of 11.1 events per year to 0.4, with nine out of ten patients experiencing no bleeding episodes over the course of the trial.
Read the full article here.
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