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PARKINSN  October 1997, Week 5

PARKINSN October 1997, Week 5

Subject:

Another promising development

From:

John Stafford <[log in to unmask]>

Reply-To:

Parkinson's Information Exchange <[log in to unmask]>

Date:

Tue, 28 Oct 1997 22:20:20 -0500

Content-Type:

text/plain

Parts/Attachments:

Parts/Attachments

text/plain (138 lines)

 Avigen Demonstrates Gene Therapy
 Success in Treating Animal Model of
 Parkinson`s Disease

 October 28, 1997

 ALAMEDA, Calif., Oct. 27 /PRNewswire/ via Individual Inc. -- Avigen (Nasdaq:
 AVGN) scientists and their collaborators in Japan announced the successful
 application of their adeno-associated virus (AAV) vector approach to gene
therapy
 for use in the treatment of Parkinson's disease in an animal model of that
illness.
 The scientists demonstrated the use of AAV vectors to deliver genes
encoding two
 enzymes involved in dopamine production into the brains of rats with an
induced
 form of Parkinson's disease. Abnormal rotational movement decreased
 significantly in parkinsonian rats receiving one or both genes, with those
animals
 receiving both genes showing greater improvement over rats receiving only
one of
 the genes.

 The new research was presented today by Avigen scientists and the Company's
 collaborators at Jichi Medical School and Fujita Health University at the
27th
 Annual Society for Neuroscience Meeting in New Orleans, Louisiana.

 Parkinson's disease is a progressive, degenerative disorder characterized
by tremor
 and rigidity, and often, slowed and abnormal movement. An estimated 500,000
 Americans suffer from the disease. The symptoms result from the progressive
 loss of brain cells that produce a neurotransmitter called dopamine which is
 released in an area of the brain called the striatum; the severity of
Parkinson's
 disease in an affected individual is proportional to the decline in the
amount of
 dopamine in their striatum.

 The brain normally produces dopamine from a precursor, the amino acid called
 tyrosine, which is a component of foods. This conversion process has two
steps.
 Tyrosine is first converted to a molecule called L-dopa by an enzyme in
the brain
 called tyrosine hydroxylase (TH); L-dopa is subsequently converted into
dopamine
 by a second brain enzyme called L-amino acid decarboxylase (AADC). Patients
 with Parkinson's disease can initially be treated by orally administering the
 intermediate, L-dopa. This treatment, however, usually becomes less effective
 with the progression of disease and must often be discontinued due to
numerous
 serious side effects produced by the increasing doses of drug that must be
 administered to achieve a therapeutic effect. A more recent experimental
approach
 has been to locally produce dopamine in the striatum by grafting fetal
midbrain
 cells into the patient's brain. There are serious practical and ethical
issues
 associated with this approach. Using gene therapy to restore the brain's
capacity to
 produce dopamine offers a novel potential alternative to the treatment of
 Parkinson's disease.

 Avigen scientists and their collaborators first showed, using a marker
gene, that
 delivery of AAV vectors containing that gene into the striatum of rats
resulted in
 gene expression for several months with no pathological changes in the
brains of
 the animals. The researchers then produced two AAV vectors, one containing
the
 TH gene (AAV-TH) and the other containing the AADC gene (AAV-AADC), and
 delivered these vectors directly into the striatum of rats having a
 laboratory-induced form of Parkinson's disease. Abnormal movement decreased
 significantly in animals receiving AAV-TH alone or a combination of AAV-TH
and
 AAV-AADC; animals receiving both vectors showed better recovery than animals
 receiving AAV-TH alone.

 "This study demonstrates the ability of AAV vectors to efficiently
transfer more
 than one gene to localized regions of the brain, and once there, to achieve
 long-term production of a therapeutic protein," said Gary Kurtzman, M.D.,
Avigen
 vice president of research and development. "Our findings offer a novel
approach
 to the treatment of Parkinson's disease, and we are evaluating the
delivery of
 additional genes to the brain that might further increase the therapeutic
effect
 observed in this study."

 "Parkinson's disease is a particularly attractive target for gene therapy
because,
 unlike many neurological disorders, the particular defect underlying the
disease and
 area of the brain affected is well understood," said John Monahan, Ph.D.,
 president and chief executive officer of Avigen. "We are very encouraged
by our
 research results to date and are looking to work with additional
collaborators and a
 corporate partner to bring this therapeutic approach to the clinic."

 Avigen, Inc. is a biotechnology company involved in the development of gene
 therapy products derived from adeno-associated virus for the treatment of
 inherited and acquired diseases. The Company's proposed gene therapy products
 are designed for in vivo administration to achieve the production of
therapeutic
 proteins within the body. Avigen is focusing its efforts on developing
products to
 deliver genes for the treatment of hemophilia, brain cancer, hyperlipidemia,
 metabolic storage diseases and blood cell-related diseases including
sickle cell
 anemia, beta-thalassemia and HIV.

 Except for the historical information contained herein, this news release
contains
 forward-looking statements that involve risks and uncertainties, including
the
 Company's preclinical and clinical efforts, the anticipated timing of
regulatory
 filings and approvals, and other risks detailed from time to time in
documents filed
 by Avigen with the SEC, including the report on Form 10-K for the year ended
 June 30, 1997 and the report on Form 10-Q for the quarterly period ended
March
 31, 1997.

 SOURCE Avigen, Inc.

 /CONTACT: Thomas J. Paulson of Avigen, Inc., 510-748-7150; or Joan
 Kureczka, 415-821-2413, or Kate Alderete, 415-355-3692, both of J. Kureczka
 Associates, for Avigen, Inc./ (AVGN)

John Stafford -- http:/www2.netcom.com/~johnws/index.htm --
[log in to unmask]
................... Si fallatis officium, quaestor infinitius eat se
quicquam scire de factis vestris.

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March 2003, Week 1
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February 2003, Week 1
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December 2002, Week 5
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June 2002, Week 5
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March 2001, Week 3
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February 2001, Week 1
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January 2001, Week 1
December 2000, Week 5
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December 1999, Week 5
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December 1998, Week 5
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February 1998, Week 1
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December 1997, Week 5
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December 1997, Week 3
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December 1995, Week 5
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