Targeted Genetics Acquires Rights to siRNA Development Program in Huntington's Disease From Sirna Therapeutics
07 4월 2008 - 8:30PM
Marketwired
SEATTLE, WA today announced that it has acquired full exclusive
rights to its preclinical Huntington's disease (HD) program from
Sirna Therapeutics, a wholly owned subsidiary of Merck & Co.,
Inc.
In exchange for these rights, which include a license to
intellectual property (IP) that Targeted Genetics may find
necessary to develop and commercialize an HD product, Targeted
Genetics will pay Sirna an undisclosed royalty on future sales.
Sirna has also assigned to Targeted Genetics a licensing agreement
it has with the University of Iowa that covers certain IP developed
by Dr. Beverly Davidson's laboratory related to RNA interference
(RNAi) including Adeno-Associated Virus (AAV) expressed RNAi.
In 2005, Targeted Genetics and Sirna Therapeutics formed a
collaboration to develop HD therapeutics using an AAV delivered
RNAi approach to target the HD gene. This collaboration also
involved Dr. Davidson's laboratory and was based on preclinical
proof of concept established by University of Iowa researchers. AAV
vectors express for long periods of time and allow for infrequent
dosing, which is highly desirable when administering a therapeutic
directly to the brain.
"This moves all of the key pieces of this program to Targeted
Genetics and gives us exclusive rights and direct involvement with
the University of Iowa to expedite the program," said H. Stewart
Parker, president and chief executive officer of Targeted Genetics.
"This program is our primary proof of concept in the area of
expressed RNAi, which we believe could present multiple product
opportunities to help patients who currently have little hope for
treatment, such as those with HD."
"The results we have produced in animal models of HD are very
exciting. We have identified and are currently evaluating lead
candidates in preclinical studies to move forward into clinical
trials," stated Beverly L. Davidson, Ph.D., Roy J. Carver professor
of medicine and vice chair of research in the Department of
Internal Medicine at the University of Iowa. "Targeted Genetics'
approach of using AAV vectors for the delivery of expressed RNAi
may have advantages over alternative RNAi delivery approaches due
to AAV's proven long-term expression capabilities, stability and
safety profile."
HD is a devastating, inherited, neurodegenerative disorder that
results from a mutation in the gene that codes for the huntingtin
protein. HD generally shows onset in mid-life and, according to the
Huntington's Disease Society of America, one of out of every 10,000
Americans has HD and an additional 200,000 are at risk of onset.
The disease-causing gene produces a defective huntingtin protein
that is toxic to certain brain cells and the subsequent neuronal
damage leads to the movement disorders, psychiatric disturbances
and cognitive decline that characterize this disease.
"There is a serious unmet medical need for an effective
treatment for HD," said Alan Sachs, M.D., Ph.D., vice president RNA
therapeutics, Merck Research Laboratories. "This agreement provides
Targeted Genetics with the freedom to advance this AAV vector
program in the hope of developing a treatment that targets the
genetic cause of this devastating condition, not just the
symptoms."
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 that encode proteins to
increase gene function or 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 Targeted Genetics' website at www.targetedgenetics.com.
Safe Harbor Statement under the Private Securities Litigation
Reform Act of 1995:
This release contains forward-looking statements regarding the
Company's liquidity and financial resources, its ability to fund
ongoing and future operations, its business strategy and product
development, including statements regarding the data collected in
the HD program, the timing, continuance or results of trials
related to the HD program, the potential impact of the HD program
on our results of operations and other statements about the
Company's 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 actual future events or results include, but are not
limited to, payments anticipated by the Company under product
development collaborations and contracts, the Company's actual
expenses, the Company's ability to raise capital when needed, the
timing, nature and results of the Company's clinical trials,
potential development of alternative technologies or more effective
products by competitors, the Company's ability to obtain and
maintain regulatory or institutional approvals, the Company's
ability to maintain its listing on the NASDAQ Capital Market and
the Company's ability to obtain, maintain and protect its
intellectual property, as well as other risk factors described in
its filings with the Securities and Exchange Commission (SEC),
including in "Item 1A. Risk Factors" in the Company's most recent
annual report on Form 10-K for the year ended December 31, 2007
filed with the SEC. You should not rely unduly on these
forward-looking statements, which apply only as of the date of this
release. The Company undertakes no duty to publicly announce or
report revisions to these statements as new information becomes
available that may change the Company's expectations.
Investor and Media Contact: Stacie D. Byars WeissComm Partners
415.946.1072 Email Contact Carolyn Wang WeissComm Partners
415.946.1065 Email Contact
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