SOURCE: Targeted Genetics

October 04, 2007 07:30 ET

Targeted Genetics Receives Milestone Payment From Amsterdam Molecular Therapeutics

SEATTLE, WA--(Marketwire - October 4, 2007) - Targeted Genetics Corporation (NASDAQ: TGEN) today announced that it has received a milestone payment from Amsterdam Molecular Therapeutics B.V. (AMT). The payment was made under a licensing agreement that provides AMT with non-exclusive rights to patents covering Adeno-Associated Virus type 1 (AAV1), a serotype of AAV with potential application in the development and commercialization of therapeutic products for the treatment of lipoprotein lipase (LPL) deficiency. AMT recently reported the initiation of a pre-registration clinical trial in Canada for AMT-011, an AAV1-based therapy for LPL deficiency, and anticipates commercial roll-out of the product in 2009. LPL deficiency in humans is a severe and debilitating disease associated with extremely high serum triglyceride concentrations, high morbidity, and increased mortality. The amount of the milestone payment is not disclosed.

"This milestone payment provides continuing validation of our strategy to leverage our intellectual property as an industry leader in AAV technology," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "As AMT advances its LPL development programs, we will realize value through potential future milestones and royalties on product sales. This strategy provides us with incremental revenue from our broad and deep patent estate, even as we stay focused on our core product development programs in inflammatory arthritis, AIDS prophylaxis, congestive heart failure and Huntington's disease."

As announced in December 2006, the non-exclusive license to U.S. patents #6,759,237 and #7,105,345, which were issued to the University of Pennsylvania and exclusively licensed to Targeted Genetics, provides AMT with the rights to use AAV1 in the development and commercialization of therapeutic products for treating type I and type V LPL deficiencies.

About LPL Deficiency

LPL deficiency is caused by a deficiency or loss of LPL, the principal enzyme involved in the clearance of triglycerides from the bloodstream. Affected patients develop chronic pancreatitis, ultimately resulting in diabetes mellitus and sometimes in death. The dysregulation in lipid metabolism that occurs in LPL deficiency also predisposes affected patients to cardiovascular diseases, including coronary artery disease and heart attack. Currently, the only treatment for the condition is a severe reduction in the consumption of dietary fat, a regimen to which many patients have difficulty adhering. AMT estimates the worldwide population of LPL deficiency at 6,000 - 8,000 patients and LPL type V deficiency at approximately 100,000.

About Targeted Genetics

Targeted Genetics Corporation is a biotechnology company committed to the development of innovative targeted molecular therapies for the prevention and treatment of acquired and inherited diseases with significant unmet medical need. Targeted Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows it to deliver genes coding proteins to increase gene function, as well as RNAi to decrease or silence gene function. Targeted Genetics' product development efforts target inflammatory arthritis, AIDS prophylaxis, congestive heart failure and Huntington's disease. To learn more about Targeted Genetics, visit the Company's website at

About AMT

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. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements regarding our licensee's ability to develop, manufacture, conduct clinical trials, accurately interpret clinical results, obtain regulatory approvals and commercialize any therapeutic products covered under the license agreement, the market prospects for any therapeutic products covered under the license agreement, our benefits, including milestone and royalty payments, that we expect to derive as a result of this license, and other statements about our plans, objectives, intentions and expectations. These statements, involve current expectations, forecasts of future events and other statements that are not historical facts. Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of forward-looking statements. Factors that could affect our actual results include, but are not limited to, our ability to obtain, maintain and protect our intellectual property, our ability to raise capital when needed, our ability to recruit and enroll suitable trial participants, the timing, nature and results of research and clinical trials, potential development of alternative technologies or more effective processes by competitors, and, our ability to obtain and maintain regulatory or institutional approvals, as well as other risk factors described in Item 1A. Risk Factors in our report on Form 10-K for the year ended December 31, 2006 and updated in Item 1A. Risk Factors in our Form 10-Q for the quarter ended June 30, 2007. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. We undertake no duty to publicly announce or report revisions to these statements as new information becomes available that may change our expectations.

Contact Information

  • Investor and Media Contacts:
    Stacie D. Byars
    Director, Communications
    Targeted Genetics Corporation
    (206) 521-7392 (office)