Greffex Gene Therapy Vector GREF8™
Hemophilia A is a bleeding disorder caused by a genetic defect of the coagulation factor VIII (F8). It is an x-linked recessive disease that affects about 1-in-5,000-to-10,000 males (approximately 40,000 in the US and a similar number in the European Union). It is characterized by bleeding into soft tissues, muscles and weight-bearing joints. Sixty percent of hemophiliacs suffer from a severe form of the disease with F8 activities of <1%. Protein replacement therapy is required continuously for severe hemophiliacs and on a need basis for the other afflicted.
As an alternative to protein replacement, F8 gene therapy has been investigated. The success of gene therapy has been hampered primarily by issues with gene therapy vectors. Due to the large size of the F8 gene, vectors based on the Adenovirus Associated Virus (AAV) are not able to deliver the entire gene. Greffex’s’ GREVAX™ vectors are devoid of Ad genes, rarely show chromosomal integration and possess payloads large enough to accommodate the F8 gene in its entirety.
In addition, Greffex’s GREF8™ vectors are charged with an immune inhibitory moiety to suppress the development of inhibitory antibodies.