First gene therapy for severe hemophilia

A hereditary disease exposing to potentially fatal bleeding, severe hemophilia A affects one in 12,000 boys in France.

Due to a genetic abnormality, these patients do not produce factor VIII (FVIII), a protein essential for blood clotting.

Current treatments make it possible to significantly improve their state of health, but at the cost of very frequent injections of FVIII throughout life.

Hence the interest aroused by the first gene therapy, recently authorized in adults by the European Medicines Agency (EMA), the effects of which can last for several years.

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Roctavian (valoctocogene roxaparvovec), a drug from the American laboratory BioMarin, makes it possible to deliver the gene coding for factor VIII to the liver cells, by means of an inactivated adenovirus (AAV5).

Only one injection is necessary and the effect begins about fifteen days later.

For patients in whom the therapy works,

reports Professor Hervé Chambost, responsible…

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