Targeted Genetics' Collaborators Present Preclinical Data in
Huntington's Disease and AAV Expression of Large Proteins at the 8th Annual
Meeting of the American Society of Gene Therapy
Presentations Demonstrate Broad Potential of Company's AAV Technology
ST. LOUIS and SEATTLE, June 2 /PRNewswire-FirstCall/ -- Targeted Genetics
Corporation (NASDAQ:TGEN) announces that its academic collaborators will
present results of preclinical studies of adeno-associated virus (AAV) vectors
in models of Huntington's disease (HD) and for expression of large proteins
today in two sessions during the 8th Annual Meeting of the American Society of
Gene Therapy in St. Louis, MO. These presentations underscore the potential
therapeutic utility of AAV vectors.
Dr. Beverly Davidson of the University of Iowa will present encouraging
preclinical results from a study of a potential treatment of HD in an oral
abstract session for neurodegenerative diseases titled, "AAV-Delivered RNAi
Improves Cellular and Motor Phenotypes in a Mouse Model for Huntington's
Disease (Abstract #52)." Dr. Davidson is an academic collaborator of Sirna
Therapeutics (NASDAQ:RNAI), Targeted Genetics' partner for the development of
novel AAV-based HD therapies. Results demonstrate that RNA interference (RNAi)
therapy may have a beneficial impact on the symptoms and progression of HD. In
the study, AAV vectors were used to deliver DNA sequences encoding short
interfering RNAs (siRNA) by direct injection into the brains of mice with HD. The studies were conducted using AAV serotype 1 (AAV1) vectors. AAV1 vectors
may enter cells in the neurological system more effectively than vectors based
on other serotypes.
In a separate poster presentation, Dr. John Engelhardt, Professor, Department
of Anatomy and Cell Biology at the University of Iowa Carver College of
Medicine and Director for the UI Center for Gene Therapy, will present Abstract
#116, titled, "Functional Comparison of Four CFTR rAAV Vectors with Alternative
Transcriptional Elements in Cell Lines and Polarized Human CF Airway
Epithelia." In this study, different regulatory elements within AAV serotype 2
vectors were compared to determine their ability to support expression of
delivered genes. This study demonstrates that AAV vectors may be optimized
based on the size of the delivered gene.
"Today's presentations highlight the growing body of data supporting the broad
utility of our AAV vector technology in the treatment of important diseases,"
said H. Stewart Parker, President and Chief Executive Officer of Targeted
Genetics. "We are very excited to be working in partnership with top
researchers from the University of Iowa toward the development of an
siRNA-based therapeutic for Huntington's disease, and the potential expansion
of our technology platform to support novel approaches to treating a variety of
diseases.
For more information about the 8th Annual Meeting of the American Society of
Gene Therapy, please refer to http://www.asgt.org/.
About Targeted Genetics Targeted Genetics Corporation develops molecular medicines for the prevention
and treatment of acquired and inherited diseases. The Company has clinical
product development programs targeting AIDS prophylaxis and inflammatory
arthritis. The Company also has a promising pipeline of preclinical product
development programs focused on hyperlipidemia, congestive heart failure, and
Huntington's disease. For more information about Targeted Genetics, visit its
website at http://www.targetedgenetics.com/.
Safe Harbor Statement under the Private Securities Litigation Reform Act
of 1995:
This release contains forward-looking statements regarding our intellectual
property, research programs and clinical trials, our product development and
our potential development platforms including AAV vectors and other statements
about our plans, objectives, intentions and expectations. In particular, the
statements regarding the Company's pipeline and potential application of this
patent to any future product candidates are forward-looking statements. 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, results of animal research are not necessarily indicative of
results in humans, the timing, nature and results of our research, potential
development of alternative technologies or more effective products by
competitors, our ability to obtain and maintain regulatory or institutional
approvals, our ability to obtain, maintain and protect our intellectual
property and our ability to raise capital when needed, as well as other risk
factors described in the section entitled "Factors Affecting Our Operating
Results, Our Business and Our Stock Price" in our Quarterly Report on Form 10-Q
for the period ended March 31, 2005. 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: Stacie D. Byars, Director, Communications of Targeted Genetics
Corporation, +1-206-521-7392. DATASOURCE: Targeted Genetics Corporation CONTACT: Stacie D. Byars, Director, Communications of Targeted Genetics Corporation, +1-206-521-7392 Web site: http://www.asgt.org/ Web site: http://www.targetedgenetics.com/
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