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 /EMWNews-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

http://www.stjude.org.



    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.