Bayer HealthCare and Dimension Therapeutics have entered into collaboration for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. Dimension specializes in the development of adeno-associated viruses (AAVs) for the purpose of gene therapy for rare diseases.
In hemophilia A, AAVs act as delivery vehicles, or vectors, to carry the genes that trigger the production of the factor VIII (VIII) protein, which is deficient in people with this disorder. AAVs can actually deliver the genetic material into living cells to sustain therapeutic effect without causing disease or triggering significant immune responses. In addition, they often target liver cells, which manufacture factor VIII.
“Currently available replacement therapies for hemophilia A are often administered intravenously multiple times a week and may be required for life, depending on the severity of a patient’s disease,” said Thomas R. Beck, MD, chief executive officer of Dimension Therapeutics. “Gene therapy offers the potential to transform the treatment of hemophilia by inserting a correct version of the faulty gene responsible for the disease. We are proud to partner with Bayer, a leader in the treatment of hemophilia A, to develop a therapy with the potential to significantly change the treatment landscape.”
Source: Bayer news release dated June 23, 2014
