Amit Nathwani, M.D., Ph.D., who initiated his work on Factor IX while in Nienhuis' lab, has continued to collaborate with St. Jude faculty Andrew Davidoff, M.D., and John Gray, Ph.D., in developing a novel AAV vector that produces therapeutic levels of Factor IX when given intravenously in animal models. This vector has been licensed to AMT.
Repair factor IX production and blood clotting
Hemophilia B is characterized by defective blood clotting due to an absence of functional clotting Factor IX. The gene with the information for the Factor IX protein is mutated in Hemophilia patients, resulting in the production of non-functional protein. AMT uses gene therapy to introduce the functional gene into the patient's cells and thereby restore blood clotting.
AMT recently acquired a license from 'TIGET' San Raffaele Telethon Institute For Gene Therapy, Italy, to use its micro-RNA technology to prevent immune responses against gene therapy for Hemophilia B. AMT will use the TIGET technology to prevent an immune response against the newly formed Factor IX, which the patient's body may recognize as being foreign.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially the ones that are caused by one faulty gene. AMT currently has a product pipeline with seven products at different stages of development.
About St Jude Children's Research Hospital
St. Jude Children's Research Hospital is internationally recogn
|SOURCE Amsterdam Molecular Therapeutics B.V|
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