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.

Alnylam second-quarter revenues increase to $26.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter ended June 30, 2010, and company highlights.

5 Aug 2010

SurModics, EGEN continue collaboration on long-term controlled release of siRNA complexes

SurModics, Inc., a leading provider of drug delivery and surface modification technologies to the healthcare industry and EGEN, Inc., a privately held biopharmaceutical company focused on developing nucleic acid (DNA and RNAi) therapeutics for the treatment of human diseases, are pleased to announce they have executed a continuation of their feasibility collaboration focused on long-term controlled release of siRNA complexes.

28 Jul 2010

RXi Pharmaceuticals receives SBIR grant to further develop novel RNAi therapeutics

RXi Pharmaceuticals Corporation, a recognized leader in RNAi-based therapeutic discovery and development, today announced that it was awarded an Advanced Technology Small Business Innovation Research grant by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health.

13 Jul 2010

Silence strengthens RNAi IP portfolio with issuance of key patent in structural modification technology

Silence Therapeutics plc announces the issuance of United States patent 7,750,144, titled Methods and Compositions for Enhancing Efficacy and Specificity of RNA Silencing, by the United States Patent and Trademark Office (USPTO). The issued patent generally claims methods of producing double stranded (RNAi) agents having decreased off-target silencing activity through certain structural modifications. The ability to minimize the off-target effects of RNAi therapeutics is critical for controlling unwanted cellular activity and/or potential safety concerns.

8 Jul 2010

Alnylam Pharmaceuticals initiates dosing in Phase I human clinical study with ALN-TTR01

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today it has initiated dosing in a Phase I human clinical study with ALN-TTR01. The study is aimed at evaluating the safety and tolerability of ALN-TTR01 in patients with transthyretin -mediated amyloidosis, and is also designed to provide preliminary data on human proof of concept based on measurements of TTR serum levels.

7 Jul 2010

Sirnaomics receives NIH SBIR grants to advance siRNA therapeutics for critical human diseases

Sirnaomics, Inc. announced today that the company has received two SBIR (Small Business Innovation Research) grants from National Cancer Institute (NCI) and National Institute of Allergy and Infectious Diseases (NIAID), of National Institute of Health (NIH). Sirnaomics, Inc., a biopharmaceutical company founded in early 2007, is dedicated to developing RNA interference (RNAi) therapeutics for treatment of critical human diseases.

30 Jun 2010

Alnylam receives Portuguese, Swedish, British approvals to initiate ALN-TTR01 Phase I study in ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that its applications for ALN-TTR01 have been given clearance by Portuguese, Swedish, and British regulatory authorities to begin clinical testing. The trial will begin enrolling patients shortly in a blinded, randomized, placebo-controlled, multicenter Phase I study.

29 Jun 2010

BIND Biosciences announces $12.4 million Series C-1 financing

BIND Biosciences, Inc. (BIND), a privately held biopharmaceutical company developing best in class therapeutics based on its proprietary targeted nanotechnology platform, announced today that it has secured a $12.4 Million Series C-1 financing. This financing included all of BIND's current investors (including Polaris Venture Partners, Flagship Ventures, ARCH Venture Partners, NanoDimension and DHK Investments) as well as a new venture investor, Endeavour Vision, and private investors.

29 Jun 2010

Alnylam presents data on novel delivery lipids at IBC 6th Annual Cell Line Development and Engineering conference

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the presentation of new data at the IBC 6th Annual Cell Line Development and Engineering conference held in San Francisco, California from June 21 - 24, 2010.

24 Jun 2010

Update on Regulus and sanofi-aventis collaboration

Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. announced today that, as a result of the newly formed alliance Regulus has established with sanofi-aventis, each company will receive a payment of $1.8 million from Regulus. This represents 7.5% of the $25 million upfront payment from sanofi-aventis to Regulus.

23 Jun 2010

Regulus Therapeutics, sanofi-aventis partner to develop and commercialize microRNA therapeutics

Regulus Therapeutics Inc. and sanofi-aventis announced today that they have entered into a global, strategic alliance to discover, develop, and commercialize microRNA therapeutics. The alliance represents the largest microRNA partnership formed to date, valued at potentially over $750 million, and includes a $25 million upfront fee, a $10 million future equity investment subject to mutual agreement on company valuation, and annual research support for three years with the option to extend two additional years.

22 Jun 2010

USPTO awards Alnylam Notice of Allowance for patent covering methods of inhibiting expression of mutant genes

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has awarded a Notice of Allowance for the "John et al." patent (Application No. 10/384,463), which covers methods of inhibiting mutant genes with double-stranded RNAs from 19 to 30 nucleotides in length.

18 Jun 2010

Mount Sinai School of Medicine, Alnylam collaborate to develop microRNA-based attenuated influenza viruses

Mount Sinai School of Medicine has completed an exclusive option agreement with Alnylam Pharmaceuticals, Inc. a leading RNAi therapeutics company, for intellectual property related to RNAi applications in vaccine development. These new applications of RNAi technology define opportunities for the advancement of novel vaccines in infectious disease.

18 Jun 2010

USPTO issues patent covering methods for enhancing RNA silencing activity of RNAi agents

Silence Therapeutics plc announces the issuance of United States patent 7,732,593, titled Methods and Compositions For Controlling Efficacy of RNA Silencing, by the United States Patent and Trademark Office. The issued patent, which is based on the seminal research of Phillip D. Zamore, Ph.D., Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair of Biomedical Sciences, and Professor of Biochemistry & Molecular Pharmacology at University of Massachusetts Medical School, generally claims methods of enhancing the RNA silencing activity of RNA interference (RNAi) agents through certain structural modifications.

14 Jun 2010

RXi announces new RNAi therapeutic strategy at Jefferies 2010 Global Life Sciences Conference

RXi Pharmaceuticals Corporation, a recognized leader in RNAi-based therapeutic discovery and development, today announced the company's new focused RNAi therapeutic strategy at the Jefferies 2010 Global Life Sciences Conference.

11 Jun 2010

Alnylam presents interim data from ALN-VSP02 Phase I trial for liver cancer at ASCO 2010

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today preliminary clinical data from its ongoing Phase I trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement.

7 Jun 2010

Royal Philips Electronics, RXi Pharmaceuticals sign joint research agreement

Royal Philips Electronics and RXi Pharmaceuticals Corporation, a US-based biopharmaceutical company, today announced that they have entered into a joint research agreement to explore the benefits of combining proprietary technologies from both companies for the targeted delivery of experimental therapeutics based on RNA interference.

4 Jun 2010

Alnylam, Mount Sinai scientists discover svRNAs that play a critical role in influenza A virus replication

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Proceedings of the National Academy of Sciences (PNAS) by Alnylam scientists and collaborators from Mount Sinai School of Medicine. In the new study, Alnylam and Mount Sinai scientists discovered a novel class of virus-produced small non-coding RNAs, called small viral RNAs (svRNAs), which play a critical role in the replication of influenza A virus.

2 Jun 2010

Study demonstrates protection against Ebola infection in nonhuman primates using RNAi

Scientists using tiny particles of genetic material to interfere in the replication process of the deadly Ebola virus have successfully prevented monkeys exposed to that virus from dying of hemorrhagic fever. The proof-of-concept study, published in this week's issue of The Lancet, suggests that such protection also should be possible in humans.

29 May 2010

Silence Therapeutics deepens diverse portfolio of RNAi intellectual property

Silence Therapeutics plc announces the issuance of United States patent 7,723,316, titled Compositions and Methods of RNAi Therapeutics For Treatment of Cancer and Other Neovascularization Disease, by the United States Patent and Trademark Office (USPTO).

26 May 2010