GENETIC THERAPY, INC./GENETICS INSTITUTE HEMOPHILIA GENE THERAPY COLLABORATION
Executive Summary
GENETIC THERAPY, INC./GENETICS INSTITUTE HEMOPHILIA GENE THERAPY COLLABORATION will make use of the blood clotting products Factor VIII and Factor IX and viral vector systems for genetic therapies for the inherited blood disorders hemophilia A and the more rare hemophilia B. Under terms of a limited exclusive R&D collaboration and licensing agreement announced Sept. 24, Genetic Therapy, Inc. (GTI) will use Genetics Institute's Factor VIII and its own viral vector systems to commercialize a product for hemophilia A. In January 1992, GTI obtained exclusive rights from the British Technology Group USA for Factor IX technology in viral and retroviral vectors for hemophilia B. GTI gains sole North American marketing rights to the Factor VIII and IX gene therapy products and worldwide manufacturing rights. Genetics Institute will have rights to the commercialized products outside of North America. Financial terms of the deal were not disclosed but no equity investment is being made by either partner. GTI's role in the collaboration includes in-house R&D and joint efforts with the National Institutes of Health "and other institutions to develop vectors containing genes that can produce missing or defective clotting proteins," company CEO James Barrett, PhD, noted. Gaithersburg, Maryland-based GTI works closely with gene therapy pioneer W. French Anderson, MD, who recently left NIH to move to the University of Southern California. Anderson is the lead researcher for the five Cooperative Research and Development Agreements GTI has with NIH. GTI has agreed to fund USC gene therapy research with $30 mil. earmarked for Anderson's new lab there ("The Pink Sheet" Aug. 31, T&G-7). Commenting on the collaboration, Genetics Institute Exec VP Patrick Gage said the deal "strengthens Genetics Institute's presence in the field of blood coagulation therapy by effectively leveraging our DNA technology and established patent estate." The company's biosynthetic antihemophilic Factor VIII product Recombinate, which will be marketed by Baxter's Hyland Division, is awaiting approval in the U.S. and Europe. The PLA was filed in May 1990 and an FDA advisory committee recommended the product for approval in December 1991 ("The Pink Sheet" Dec. 16, 1991, p. 14). Genetics Institute will manufacture Recombinate.