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SOURCE: UroGen Corp
UroGen Receives FDA Clearance to Enter Human Clinical Trial Of Gene Therapy for Hemophilia A
Key Preclinical Studies Recently Published in BLOOD
SAN DIEGO, Feb. 11 /PRNewswire/ -- UroGen Corp (OTC Bulletin Board: UROG - news) today announced that the U.S. Food and Drug Administration (FDA) cleared the Company's Investigational New Drug (IND) application for a Phase I human clinical trial of its Factor VIII gene therapy product for the treatment of hemophilia A. The Company expects to begin the clinical trial later this year, following review of the clinical trial protocol by the National Institutes of Health Recombinant DNA Advisory Committee.
Research supporting the approval was published in the February 1 issue of BLOOD, the Journal of the American Society of Hematology. These studies, performed in mice with hemophilia A, a deficiency in the clotting protein Factor VIII, demonstrated sustained production of therapeutic levels of Factor VIII following a single injection of UroGen's gene therapy product. The expression of the Factor VIII protein continued for approximately one year following the injection, in effect, correcting the abnormality.
``FDA clearance of this IND application is a significant milestone in the development of our gene therapy product for hemophilia A, as well as a major milestone for the Company,' said Robert E. Sobol, M.D., president and chief executive officer of UroGen. ``We look forward to initiating our human clinical studies, which build on the promising foundation of our pre-clinical research.'
UroGen's gene therapy product for hemophilia A is an improved gene delivery system derived from the common cold virus. In contrast to earlier gene delivery approaches, UroGen's product has been genetically modified to replace all the genes responsible for transmitting the virus with therapeutic genes that are responsible for the production of Factor VIII. This product carries the entire human Factor VIII gene sequence and is designed to result in production of Factor VIII in the liver, one of its normal biological sites of production.
Hemophilia A is a hereditary disease characterized by a deficiency in the blood clotting protein Factor VIII. The disorder results in abnormal bleeding and affects approximately 20,000 individuals in the U.S.
UroGen is a biotechnology Company developing gene therapy products for the treatment of hemophilia A and prostate cancer. The Company's hemophilia product is being developed in collaboration with Baxter Healthcare. Its prostate cancer product development program has been supported, in part, by a grant from the National Cancer Institute. The Company recently announced the completion of an $8.3 million equity financing, which will contribute additional financial resources to the development of these products. |
