Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.

Hemophilia A is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Symptoms will vary and are dependent on the level factor VIII present but in the more severe cases, excessive bleeding and severe pain are common symptoms. Treatment for hemophilia is factor VIII replenishment. Currently, most hemophilia A patients take a recombinant factor VIII product to control bleeding and numerous products are available, each with its own set of benefits and challenges.

One potential treatment for these patients may be gene therapy. At the 61st American Society of Hematology (ASH) Annual Meeting & Exposition, data from an ongoing phase I/II study was presented and showed that Bayer’s gene therapy appears to be safe and effective in the first two persons that have received treatment with BAY 2599023, a gene therapy that uses a modified form of adeno-associated virus (AAV), called AAVhu37, to deliver a functional copy of the FVIII gene to liver cells.