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AMT Starts Collaboration With St. Jude Children's Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy

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 collaborating with them. "

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 recognized for its pioneering work in finding cures and saving children with cancer and other catastrophic diseases. Founded by late entertainer Danny Thomas and based in Memphis, Tennessee, St. Jude freely shares its discoveries with scientific and medical communities around the world. No family ever pays for treatments not covered by insurance, and families without insurance are never asked to pay. St. Jude is financially supported by ALSAC, its fundraising organization. For more information, please visit

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of Amsterdam Molecular Therapeutics only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.

SOURCE Amsterdam Molecular Therapeutics B.V
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