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Sirna granted patent covering siRNAs targeting Huntington's Disease.

Sirna Therapeutics, Inc. (San Francisco, CA) the leading clinical-stage RNAi therapeutics company,said that the Australian Patent Office has issued a broad patent covering vector expressed siRNAs targeting the Huntington's disease (HD) gene. This is the first patent issued anywhere in the world which encompasses 'vector expressed' siRNAs targeting a disease gene. The patent claims, which are not limited to a specific siRNA sequence or structure, broadly cover any vector expressed siRNA used against the HD gene. This patent significantly advances Sirna's position as the leader in the development of RNAi-based therapeutics for the treatment of Huntington's disease.

Entitled "siRNA-mediated gene silencing with viral vectors" (AU 2005200828), the patent further strengthens Sirna's portfolio and the company's leadership position in developing siRNA-based therapeutics. This patent includes vector expressed siRNAs with 3' -overhangs and blunt ends and short hairpin RNA (shRNA). The patent also covers methods of using siRNAs to inhibit Huntington's disease gene expression. To date, Sirna has filed over 125 patents in multiple countries around the world on the use of siRNAs targeting mammalian genes and viruses responsible for human diseases.

Bharat M. Chowrira, PhD, JD, Vice President Legal Affairs and Chief Patent Counsel of Sirna Therapeutics, stated, "Securing the broad siRNA patent for the HD gene enables Sirna to expand its dominant patent portfolio, while demonstrating the company's commitment to developing RNAi-based therapeutics for diseases that, until now, have proven difficult to treat by conventional means. In addition to demonstrating the strength of our science, today's announcement advances our strategy of obtaining broad coverage of siRNAs targeting an entire gene. It is likely that Sirna's broad siRNA claims covering an entire targeted gene, such as the HD patent, will issue worldwide, and thus prevent other companies from pursuing such targets."

The issuance of the patent follows three target patents and two broad-based siRNA patents secured this year by Sirna in the United Kingdom. The UK patents, which address the Hepatitis C Virus, Vascular Endothelial Growth Factor and Vascular Endothelial Growth Factor Receptor-1, broadly cover siRNAs against these targets. The company was also issued broad patents covering the chemical and structural modifications of siRNAs necessary for the creation of viable siRNA-based therapeutics as well as siRNAs targeting conserved regions of viruses and mammalian genes. In the coming months Sirna expects that patents with similar scope and breadth will be issued in other major countries including the U.S., Europe and Japan.

The HD patent, which is exclusively licensed to Sirna, is based on pioneering research carried out by Dr. Beverly Davidson, Ph.D., Roy J. Carver Professor in Internal Medicine Professor, Neurology and Physiology & Biophysics Vice Chair for Research, Dept of Internal Medicine Associate Director, Center for Gene Therapy University of Iowa College of Medicine. Dr. Davidson is a member of the Sirna Scientific Advisory Board and a collaborator on Sirna's efforts in HD. In 2004, Sirna partnered with Dr. Davidson and Targeted Genetics, Corp. to move forward a novel treatment for HD capitalizing on the cutting-edge work of Dr. Davidson, the novel adeno-associated viral vector (AAV) delivery technology of Targeted Genetics and Sirna's siRNA constructs. In addition, Sirna and Targeted Genetics have sponsored a joint research collaboration with Krys Bankiewicz, M.D., Ph.D., Professor of Neurological Surgery, University of California, San Francisco. Dr. Bankiewicz is developing a unique delivery mechanism to introduce AAV vector encoded siRNA constructs directly into the brain to target the HD gene.

Huntington's Disease (HD) is a devastating, inherited, degenerative brain disorder that is progressive and always fatal and for which there is, at present, no effective treatment or cure. HD affects more than 30,000 people in the United States, with another 200,000 at risk of inheriting the deadly gene. The disease-causing gene produces a 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. It is a dominant, fatal neurological disorder with no cure and no treatment that directly reduces mutant htt gene expression or otherwise prevents onset of the disease. Early in 2005, Sirna announced its selection of investigational compounds for preclinical development based on the groundbreaking work of the company's research collaborator, Dr. Beverly Davidson at the University of Iowa.

Sirna has both in-licensed patents with third parties and filed patents on technology discovered inside the company. Sirna's internal scientific work has resulted in 44 issued patents and over 250 pending patents. These patents include: 10 pending patents for siRNAs & micro-RNAs, one granted and 10 pending patents for "No-Ribo" siNAs, 5 pending patents for Multifunctional siRNAs, 23 issued and over 70 filed patents for Oligonucleotide Chemistry and Delivery, 15 issued and 25 filed patents for Oligonucleotide Manufacturing, and three granted and over 100 patents pending for fully enabled siRNAs against specific gene targets. To ensure that Sirna can bring RNAi-based therapeutics to the market, the company has pursued an in-licensing strategy to provide itself total freedom to do so in all disease areas. Sirna's in-licensing strategy has encompassed several key patents, including the Tuschl Patent from the University of Massachusetts which covers the seminal RNA interference technology covering siRNA for uses relating to human and veterinary therapeutic, prophylactic, diagnostic and healthcare applications, as well as, Carnegie Patent from the Carnegie Institution of Washington and UMASS. The Carnegie Patent is based on the pioneering work of Drs. Andrew Fire and Craig Mello in C. elegans governing genetic inhibition of genes by double-stranded RNA via RNAi.

Sirna has filed enabling patents for over 125 important mammalian disease targets including: alpha-synuclein (Parkinson's disease), HBV (hepatitis B), HCV (hepatitis C), HD (Huntington's disease), HIV (human immunodeficiency virus), HR (hairless gene), IL-4, IL-13, IL-4 Receptors, IL-13 Receptors (asthma, respiratory diseases), NOGO & NOGO Receptors (spinal cord injury), PTP-1B (diabetes, obesity), RSV (Respiratory Syncytial virus), and VEGF (angiogenesis, AMD, diabetic retinopathy, cancer, kidney disease).

Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis B and C, dermatology, asthma, Huntington's Disease, diabetes and oncology. Sirna Therapeutics has presented interim Phase 1 clinical trial data for its most advanced compound, Sirna-027, a chemically optimized siRNA targeting the clinically validated vascular endothelial growth factor pathway to treat AMD. The results to date, demonstrate that Sirna-027 for AMD is safe and well tolerated. Visual acuity has stabilized in 100 percent of patients treated and clinically significant improvement, of three lines or greater on the ETDRS eye chart, has been observed in 25 percent of the patients tested. Sirna Therapeutics has collaborations with Eli Lilly and Company, Targeted Genetics, Archemix Corporation and Protiva Biotherapeutics. Sirna has a leading intellectual property portfolio in RNAi with 43 issued patents and over 250 pending applications worldwide.

Sirna Therapeutics, Inc.

+1-303-449-6500

www.sirna.com/
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Comment:Sirna granted patent covering siRNAs targeting Huntington's Disease.
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Geographic Code:1USA
Date:Sep 1, 2005
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