Small interfering RNA (siRNA), sometimes known as short interfering RNA or silencing RNA, is a class of double-stranded RNA molecules, 20-25 nucleotides in length, that play a variety of roles in biology. Most notably, siRNA is involved in the RNA interference (RNAi) pathway, where it interferes with the expression of a specific gene. In addition to their role in the RNAi pathway, siRNAs also act in RNAi-related pathways, e.g., as an antiviral mechanism or in shaping the chromatin structure of a genome; the complexity of these pathways is only now being elucidated.
Drug discovery scientists need to broaden their approaches to pharmaceutical research and development (R&D) and Medicinal Chemistry, to take advantage of untapped opportunities, says Torsten Hoffmann, Head Discovery Chemistry at F. Hoffmann La Roche, Basel, Switzerland. Chemical Biology allows room for innovation, but is often overlooked by scientists in the industry.
Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference therapeutics, announced today that the United States Patent & Trademark Office (USPTO) and the European Patent Office (EPO) have issued key patents covering elements of Tekmira's leading lipid nanoparticle (LNP) technology, which is used to deliver nucleic acid therapeutics, including small interfering RNA (siRNA).
Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, presented new data at the MIT Center for Biomedical Innovation's Biomanufacturing Summit from its platform efforts aimed at developing RNAi technologies for applications in biologics manufacturing.
Silence Therapeutics plc announces positive research findings demonstrating the ability of the Company's lead RNA interference (RNAi) drug candidate, Atu027, to prevent the formation of pulmonary metastasis in various animal models of cancer metastasis. Researchers showed that Atu027 inhibited multiple key biological processes that enable and contribute to the dissemination and formation of pulmonary metastases in a murine breast cancer metastasis model.
Bio-Path Holdings, Inc., a biotechnology company developing a liposomal delivery technology for nucleic acid cancer drugs, today announced that it has received a grant of $244,479 from the United States Government to help fund the Company's Phase I clinical trial of its lead cancer drug candidate Liposomal Grb-2. The amount of funds awarded was the maximum allocation allowed under the program.
Alnylam Pharmaceuticals, Inc. a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter ended September 30, 2010, and company highlights.
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today it will present several poster presentations at the 61st Annual Meeting of the American Association for the Study of Liver Diseases being held in Boston, Mass from October 29 - November 2, 2010.
Techulon™ Inc., an early stage life sciences company based in the Virginia Tech Corporate Research Center, has signed an exclusive license with Virginia Tech Intellectual Properties Inc. to market a new theranostic transfection reagent. This new traceable reagent complements Techulon's currently marketed Glyocofect™ transfection platform and expands the company's offering in an $800 million market that is steadily growing at 15% annually.
Amsterdam Molecular Therapeutics (AMT) Holding N.V. a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks. These preliminary results suggest that this approach could lead to a treatment for high cholesterol in humans
Altogen Biosystems announced the release of its second generation of in vivo RNAi transfection reagent in the company's popular in vivo delivery product line: PEG-Liposome siRNA In Vivo Transfection Kit. This reagent is also compatible for in vivo transfection of other negatively charged molecules – RNA, DNA, and small proteins.
In a report published in the Oct. 20 issue of Science Translational Medicine, researchers at NewYork-Presbyterian Hospital/Weill Cornell Medical Center say animal and human data suggest gene therapy to the brain may be able to treat patients with major depression who do not respond to traditional drug treatment.
Dicerna Pharmaceuticals, Inc. a second generation RNA interference (RNAi) company developing novel therapeutics utilizing its proprietary Dicer Substrate Technology and Dicer Substrate siRNA (DsiRNA) molecules, today announced a second closing of its Series B equity financing, securing an additional $4 million and bringing the total capital raised in this round to $29 million.
Quark Pharmaceuticals, Inc., a pharmaceutical company engaged in the discovery and development of RNAi-based therapeutics, today announced it has dosed the first patient with recent onset of non-arteritic anterior ischemic optic neuropathy (NAION) in its ongoing Phase I study of QPI-1007, the Company's proprietary synthetic siRNA drug candidate for ocular neuroprotection.
Silence Therapeutics plc announces that the European Patent Office (EPO) has granted a new patent that provides broad protection on novel aspects of the RNAi structural modification technology known as the Zamore Design Rules.
FibroGen, Inc. today announced initiation of a phase 2 study to compare the efficacy of FG-3019, a human monoclonal antibody against connective tissue growth factor (CTGF), with placebo in reversing liver fibrosis (scarring of the liver) due to chronic infection with hepatitis B virus (HBV).
Scientists have found that a synthetic molecule they designed can block activation of a gene in liver cancer cells, halting a process that allows some of those cancer cells to survive chemotherapy.
Researchers at the Georgia Institute of Technology and Emory University have developed a novel approach for delivering small bits of genetic material into the body to improve the treatment of inflammatory bowel diseases. Delivering short strands of RNA into cells has become a popular research area because of its potential therapeutic applications, but how to deliver them into targeted cells in a living organism has been an obstacle.
ACCESS PHARMACEUTICALS, INC., a biopharmaceutical company leveraging its proprietary drug-delivery platforms to develop treatments in areas of oncology, cancer supportive care and diabetes, announced today that it has made significant progress with its proprietary Cobalamin-targeted drug-delivery program for siRNA therapies.
Arrowhead Research Corporation today announced that it has increased its ownership of majority owned subsidiary Calando Pharmaceuticals through the exchange of Calando Series A Preferred Stock for Arrowhead warrants.
Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that it has advanced miravirsen (SPC3649), the first microRNA-targeted drug to enter clinical trials, into Phase 2 studies to assess the safety and tolerability of the drug in treatment-naive patients infected with the Hepatitis C virus.
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