AMT Starts Collaboration With St. Jude Children's Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Fin...( AMSTERDAM The Netherlands July 15...)

AMSTERDAM, The Netherlands, July 15 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced the start of a collaboration with St. Jude Children's Research Hospital in Memphis, Tennessee, USA, on the development of a gene therapy treatment for Hemophilia B. Under the deal, AMT will receive the exclusive commercial rights to the final product. The combination of this gene with AMT's proprietary adeno-associated virus (AAV) gene therapy platform could potentially cure this seriously debilitating disease with a single administration of the product.

Hemophilia B is characterized by severe episodes of external and internal bleeding that result in significant morbidity, cause long-term damage in the joints and may be fatal when occurring in tissues like the brain. Current treatments require up to three injections a week to stop or prevent bleeding, which is a burden for young children, may be associated with abnormal blood clotting and does not prevent recurrent bleeding episodes.

Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almost always in males. The total number of patients in Europe and the US is estimated between 35,000 and 40,000.

Important collaboration with leading research institute

Under the agreement AMT will sponsor research in Hemophilia B at St. Jude. Ronald Lorijn, CEO of AMT said: "The importance of this collaboration with renowned St. Jude Children's Research Hospital stretches beyond its scientific and business aspects. This collaboration will bring to these patients the hope that a real cure is on the horizon. Access to the Factor IX gene therapy program perfectly complements our gene therapy platform allowing us to develop an effective and long-lasting therapy for Hemophilia B. Dr. Arthur W. Nienhuis and his group at St. Jude have done very important scientific work on this disease and we really look forward to collabor
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