Full Press Release Details
Genetics Corporation
GENETICS LEVERAGES VALUE OF ITS AAV PATENT ESTATE IN LICENSING AGREEMENT
AMSTERDAM MOLECULAR THERAPEUTICS
license covers the use of Targeted Genetics' AAV1 technology
the treatment of lipoprotein lipase deficiencies-
December 12, 2006 - Targeted Genetics Corporation (Nasdaq: TGEN) today announced
that the company has licensed two patents in its broad portfolio of AAV
intellectual property to Amsterdam Molecular Therapeutics B.V. (AMT). 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
lipoprotein lipase (LPL) deficiencies. AMT currently is conducting Phase
trials of AMT-011, an AAV1-based therapy for type I LPL deficiency, and
anticipates launching the product in the European Union in 2008. LPL deficiency
in humans is a severe and debilitating disease associated with extremely
serum triglyceride concentrations, high morbidity, and increased
terms of the non-exclusive license, Targeted Genetics will receive an upfront
payment from AMT of $1,750,000, as well as milestone payments and royalties
the sale of any products commercialized using the licensed technology.
agreement demonstrates our ability to leverage our portfolio of AAV-related
intellectual property assets to generate revenue for Targeted Genetics and
for our shareholders," said H. Stewart Parker, president and chief executive
officer of Targeted Genetics. "In addition, it allows us to participate,
financially, in the commercialization of AAV therapeutics by others. We are
truly seeing the expansion of interest in AAV-based molecular medicines as
therapeutic platform. AMT-011 could be the first commercially available
AAV-based therapeutic and may provide us with relatively near-term milestones
and potential royalty revenue streams to support our internal product
development efforts. We wish AMT success as they move this therapeutic candidate
through development."
uses a recombinant AAV1 vector to deliver the gene encoding lipoprotein lipase.
In preclinical studies, AMT-011 has demonstrated effective replacement of
gene function, leading to complete
normalization of triglyceride levels in two different LPL deficient animal
trial of AMT-011 in patients with type 1 LPL deficiency was initiated in
December 2005 and is ongoing.
license is a key step in AMT's commercialization strategy of its lead drug
candidate AMT-011. This breakthrough gene therapy may allow AMT to provide
patients suffering from a serious, debilitating and often life-threatening
metabolic disease, caused by a monogenetic defect, a fundamental cure", said
Ronald H.W. Lorijn, MD, PhD, M.B.A., Chief Executive Officer of
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. 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 effective
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 type 1 deficiency at 4,000 patients and LPL
V deficiency at approximately 100,000.
Genetics Corporation is a biotechnology company committed to the development
innovative targeted molecular therapies for the prevention and treatment
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
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,
the Company's website at www.targetedgenetics.com.
Molecular Therapeutics BV is a fully integrated gene therapy company founded
scientists of the University of Amsterdam Medical Center (AMC) in 1998. AMT
focuses on the development of gene-based therapies for orphan metabolic,
nervous system and ocular diseases. AMT's long-term gene expression technology
is based on specific delivery of therapeutic genes into target organs or
tissues. Its lead product, AMT-011, is in phase II for the first indication:
treatment of lipoprotein lipase deficiency type I. For further information,
visit AMT's website at www.amtbv.com
Harbor Statement under the Private Securities Litigation Reform Act of
release contains forward-looking statements regarding our licensee's ability to
develop, manufacture, 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
license agreement, our benefits, including milestone and royalty payments,
we expect to derive as a result of this license, and other statements about
plans, objectives, intentions and expectations. These statements, involve
current expectations, forecasts of future events and other statements that
not historical facts. Inaccurate assumptions and known and unknown risks
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 licensee's ability to recruit and enroll
suitable trial participants, the timing, nature and results of research and
clinical trials of our license, potential developments of alternative
technologies or more effective processes by parties other than the licensee,
and, our licensee's ability to obtain and maintain regulatory or institutional
approvals, as well as other risk factors described in "Part I, Item 1A. Risk
Factors" in our most recent annual report on Form 10-K or "Part II, Item
Risk Factors" in our most recent quarterly report on Form 10-Q filed with
Securities and Exchange Commission. You should not rely unduly on these
forward-looking statements, which apply only as of the date of this release.
undertake no duty to publicly announce or report revisions to these statements
as new information becomes available that may change our