RNAi Therapeutics News and Research

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RNA interference (RNAi) was discovered less than a decade ago and already there are human clinical trials in progress or planned. A major advantage of RNAi versus other anti-sense based approaches for therapeutic applications is that it utilizes cellular machinery that efficiently allows targeting of complementary transcripts, often resulting in highly potent down-regulation of gene expression.

EPO rules in favor of Alnylam Pharmaceuticals’ Kreutzer-Limmer '945 patent

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the Board of Appeals of the European Patent Office (EPO) has ruled that claims of the Kreutzer-Limmer '945 (EP 1214945) patent meet needed requirements under European patent law. The '945 patent comprises broad claims covering the length and certain structural requirements for siRNAs that are important for advancement of RNAi therapeutics.

20 May 2010

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of pre-clinical research in the journal Molecular Therapy revealing key mechanisms related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs).

12 May 2010

Pfizer presents data on in vivo activity of PF-04523655 siRNA drug candidate at ARVO annual meeting

Quark Pharmaceuticals, Inc., the world leader in clinical development of RNAi-based therapeutics, announced today that researchers from Pfizer, Inc. presented data on in vivo activity of PF-04523655 at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting, being held May 2-6, 2010 in Fort Lauderdale.

7 May 2010

Alnylam Pharmaceuticals reports consolidated financial results for first quarter 2010

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter ended March 31, 2010, and company highlights.

6 May 2010

Quark to present poster on predicting risk of DGF following machine perfusion of renal transplants at ATC 2010

Quark Pharmaceuticals, Inc., the world leader in clinical development of RNAi-based therapeutics, announced today the presentation of a poster at the 2010 American Transplant Congress titled, "A Nomogram for Predicting Risk of Delayed Graft Function Following Machine Perfusion of Renal Transplants."

5 May 2010

Alnylam Biotherapeutics presents new data on RNAi technologies at Cell Culture Engineering XII

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the presentation of new data at the Cell Culture Engineering XII conference held in Alberta, Canada from April 25 - 30, 2010. Alnylam Biotherapeutics was formed by Alnylam to develop RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies.

4 May 2010

Alnylam Pharmaceuticals announces EPO intent to grant notification for patent series covering RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the European Patent Office (EPO) has issued a notification of an intent to grant for the "Manoharan II" patent series (EP Application No 04 718 537), titled "Therapeutic Compositions." The new patent includes 16 claims covering compositions and methods, including pharmaceutical compositions, for chemically modified siRNAs containing phosphorothioate and 2′-O-alkyl modifications without any siRNA length restrictions.

3 May 2010

Alnylam Pharmaceuticals publishes encouraging results from Phase II GEMINI study in PNAS

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has published results from its Phase II GEMINI study in the Proceedings of the National Academy of Sciences (PNAS). These data, previously presented at the International Symposium on Respiratory Viral Infections in Singapore in 2008, showed human proof of concept with an RNAi therapeutic in a randomized, double blind, placebo-controlled study.

27 Apr 2010

Novartis exercises current right to purchase 55,223 unregistered shares of Alnylam's common stock

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that Novartis has elected to fully exercise its current right to purchase 55,223 unregistered shares of Alnylam's common stock in accordance with the terms of the Investor Rights Agreement between Alnylam and Novartis, dated September 6, 2005.

26 Apr 2010

Alnylam Pharmaceuticals presents new data on ALN-TTR01 RNAi therapeutic at amyloidosis symposium

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, presented new pre-clinical data from its ALN-TTR program at the XII International Symposium on Amyloidosis in Rome on April 18 - 21, 2010. ALN-TTR01 is a systemically delivered RNAi therapeutic being developed for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR), including familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy (FAC).

22 Apr 2010

Silence Therapeutics, AstraZeneca announce one-year extension of ongoing siRNA delivery collaboration

Silence Therapeutics plc announces that the Company and AstraZeneca have agreed to a one-year extension of their ongoing small interfering RNA (siRNA) delivery collaboration. Under this collaboration, which was originally entered into in March 2008, the companies are leveraging a number of Silence's proprietary lipid delivery and targeting technologies to develop new and enhanced delivery approaches for RNA interference (RNAi) therapeutics.

21 Apr 2010

Silence Therapeutics reorganizes resources following Intradigm merger

Silence Therapeutics plc announces that the Company is executing a strategic plan designed to optimally organize its resources following its recent merger with Intradigm Corporation. Moving forward, all research activities related to Silence's novel RNA interference (RNAi) therapeutic platforms, including technologies that have emerged from both Silence and Intradigm, will take place at the Company's Berlin location.

13 Apr 2010

USPTO allows Alnylam Pharmaceuticals' new claims from Woppmann et al. patent series

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent Office (USPTO) has allowed a patent (Application No. 10/560,336) for the Woppmann et al. patent series, entitled "Double-stranded ribonucleic acid with increased effectiveness in an organism." The newly allowed patent includes 31 claims broadly covering compositions and methods, including pharmaceutical compositions, for small interfering RNAs (siRNAs), the molecules that mediate RNAi.

8 Apr 2010

RXi Pharmaceuticals reports net loss of 28% for 2009

RXi Pharmaceuticals Corporation, a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today reported financial results for the fourth quarter and year ended December 31, 2009.

31 Mar 2010

Japanese Patent Office grants Alnylam Pharmaceuticals' claims in second patent application for Tuschl II

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the Japanese Patent Office has granted claims in a second patent application (JP Application Number 2006-317758) for the Tuschl II patent series, entitled "RNA Interference Mediating Small RNA Molecules." In May 2008, the Japanese Patent Office granted a related set of claims in the Tuschl II patent series (JP 4 095 895).

31 Mar 2010

Silence Therapeutics, Dainippon Sumitomo Pharma announce expansion of siRNA delivery collaboration

Silence Therapeutics plc today announced that the company and Dainippon Sumitomo Pharma Co., Ltd. have agreed to an expansion of their ongoing small interfering RNA (siRNA) delivery collaboration. Under this collaboration, which was originally entered into in August 2009, the companies are leveraging Silence's proprietary siRNA molecules and multiple lipid delivery and targeting technologies to demonstrate functional delivery of RNA interference (RNAi) therapeutics to specific disease targets in the body.

30 Mar 2010

Dicerna Pharmaceuticals, Ipsen announce collaboration in DsiRNA research and peptide engineering

Dicerna Pharmaceuticals, Inc., (Dicerna) a second generation RNA interference (RNAi) company, and Ipsen, a global biotechnology specialty care group, announced today that the two companies have entered into an exclusive research collaboration agreement to leverage their expertise in Dicer Substrate siRNA (DsiRNA) research and peptide engineering. The companies will develop novel conjugates of Dicerna's DsiRNA molecules and Ipsen's peptide targeting vectors in the therapeutic areas of oncology and endocrinology.

30 Mar 2010

RXi Pharmaceuticals to present advances in RNAi therapeutics at 3rd International Scar Club Meeting

RXi Pharmaceuticals Corporation, a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), announced that Pamela Pavco, Ph.D., Vice President of Pharmaceutical Development, will be presenting at the 3rd International Scar Club Meeting in Montpellier, France on March 26, 2010.

26 Mar 2010

Alnylam Pharmaceuticals earns $20M technology transfer payment from Takeda Pharmaceutical

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today announced that it has earned a $20 million technology transfer payment from Takeda Pharmaceutical Company Limited as part of the strategic alliance the companies formed in May 2008.

23 Mar 2010

Calando Pharmaceuticals demonstrates first targeted siRNA-containing nanoparticle delivery to tumors

Arrowhead Research Corporation today announced that the clinical trial being conducted by majority-owned subsidiary, Calando Pharmaceuticals, Inc., has demonstrated systemic delivery of siRNA and the successful “silencing” of a widely recognized cancer gene via RNA interference (RNAi) in humans.

22 Mar 2010