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.
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.
Molecularly targeted therapies can reduce tumors rapidly. However, not all tumors respond to the drugs, and even those that do often develop resistance over time. Looking for a way to combat the problem of resistance, researchers at Fox Chase Cancer Center hypothesized that hitting already weakened cancer cells with a second targeted agent could kill them-but only if it was the right second agent.
In order for targeted therapies against cancer to be effective, scientists need to understand upfront what related proteins in a signaling "network" makes a cancer cell resistant to a drug and selectively target them as well, say researchers at Georgetown Lombardi Comprehensive Cancer Center and Fox Chase Cancer Center.
Patients who experience physical or psychological stress - including rigorous exercise - one or two days before a cancer treatment might be unknowingly sabotaging their therapy, new research suggests.
Silence Therapeutics plc announces the issuance of two new patents by the United States Patent and Trademark Office (USPTO) which are broadly directed to double-stranded short interfering RNA (siRNA) sequences against the validated cancer targets epidermal growth factor receptor related protein (EGFR-RP) and vascular endothelial growth factor receptor 1 (VEGFR1).
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