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EXHIBIT 99.1
Sirna Therapeutics and Targeted
Genetics Form Huntington's Disease Collaboration
- Agreement Pairs Leaders in RNA Interference
(RNAi) and AAV-Based Delivery Technologies
BOULDER, Colo. and SEATTLE, Jan. 11 -- Sirna
Therapeutics, Inc. (Nasdaq: RNAI) and Targeted Genetics Corporation
(Nasdaq: TGEN) today announced that they have established a
collaboration to develop a novel therapy for the treatment of
Huntington's Disease (HD), an incurable neurodegenerative disorder.
The collaboration significantly advances Sirna's HD program by
using Targeted Genetics' adeno-associated virus (AAV) delivery
platform to develop an HD product.
The focus of the collaboration will be the
development of a therapeutic short interfering RNA (siRNA)
targeting the gene that encodes the Huntington's Disease protein.
The siRNA will be expressed from an AAV-vector. RNAi is a mechanism
used by cells to regulate the expression of genes and replication
of viruses. The RNA interference mechanism uses siRNA to induce the
destruction of target RNA using naturally occurring cellular
protein machinery. Sirna's scientific advisor and collaborator, Dr.
Beverly Davidson at the University of Iowa, has published data
demonstrating that the delivery of siRNA using an AAV vector
efficiently inhibited gene expression in an animal model of
spinocerebellar ataxia 1 (SCA1), a member of a class of inherited
human neurodegenerative diseases that includes Huntington's Disease
(Nature Medicine 10:816-820 [2004]).
"There are no treatments for Huntington's
Disease, and we believe that vector expressed RNAi-based approaches
may have significant potential in slowing or halting disease
progression," said Howard Robin, President and Chief Executive
Officer at Sirna. "Dr. Davidson's work using AAV vectors to deliver
siRNA for the treatment of related neurodegenerative disorders is
quite promising. Through the collaboration announced today we are
gaining access to Targeted Genetics' extensive capabilities in the
development, clinical testing and manufacture of AAV-based
products. We are excited to explore the potential of AAV-RNAi
therapies for Huntington's Disease, and are very pleased to be
working with the leading AAV-based product development
company."
"We are excited to continue expanding our
AAV-based gene delivery platform into the area of RNA interference
by working with Sirna, the leading company developing this
innovative technology," said H. Stewart Parker, President and Chief
Executive Officer of Targeted Genetics. "This collaboration is
consistent with our strategy to leverage our gene delivery
capabilities beyond gene replacement. Shutting down the production
of disease-causing proteins by delivering expressed RNAi sequences
complements the other applications of our technologies: providing a
functional copy of the gene deficient in cystic fibrosis,
delivering a gene encoding a therapeutic protein for rheumatoid
arthritis and stimulating the immune system using a DNA-based
vaccine against HIV."
Under the terms of the agreement, development
costs and revenue from partnering and sales of products developed
under the col
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