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Horizon, AstraZeneca partner to explore oncology-relevant genotypes to identify novel drug targets

Horizon Discovery and AstraZeneca have entered into a research, collaboration and license agreement to explore a range of oncology-relevant genotypes with the aim of identifying and validating a number of novel drug targets. This deal is the second collaboration between AstraZeneca and Horizon, and follows the announcement in April 2013 of an oncology discovery program to explore Horizon's first-in-class kinase target program, HD-001.

7 Jan 2014

Research finding could lead to development of vaccine and therapies for mucormycosis

Mucormycosis is a deadly infection that strikes people with weakened immune systems when certain types of fungi, called Mucorales, invade the patients' cells. A novel protein on the surface of the Mucorales cells, called CotH, makes this invasion possible.

22 Dec 2013

Mirimus develops enhanced tools for reversible gene suppression

Over the last decades, RNA interference (RNAi) has become an indispensable tool for functional genetic studies by harnessing the power of a cell intrinsic mechanism enabling reversible gene silencing.

17 Dec 2013

Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in development for the treatment of TTR-mediated amyloidosis.

17 Dec 2013

NIH data-sharing may advance genetic and translational research, therapeutic target discovery

For the first time, large-scale information on the biochemical makeup of small interfering RNA (siRNA) molecules is available publicly. These molecules are used in research to help scientists better understand how genes function in disease.

12 Dec 2013

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans.

10 Dec 2013

Arrowhead reports Phase 1 study data on ARC-520 for treatment of chronic hepatitis B infection

Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, today announced that COO and Head of R&D, Bruce Given, M.D., presented data on the Phase 1 clinical study of ARC-520, the company's clinical candidate for the treatment of chronic hepatitis B infection, at the HepDART 2013 conference being held on The Big Island, Hawaii.

9 Dec 2013

Arrowhead seeks approval to start ARC-520 phase 2a trial for chronic hepatitis B infection

Arrowhead Research Corporation, a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it recently filed an application for approval to begin a phase 2a clinical trial of its RNAi-based therapeutic candidate, ARC-520, for the potential treatment of chronic hepatitis B virus infection.

25 Nov 2013

Scientists use RNAi technology to reveal genes responsible for treating Parkinson's disease

Scientists at the National Institutes of Health have used RNA interference (RNAi) technology to reveal dozens of genes which may represent new therapeutic targets for treating Parkinson's disease. The findings also may be relevant to several diseases caused by damage to mitochondria, the biological power plants found in cells throughout the body.

24 Nov 2013

GoInformatics, TeselaGen form strategic partnership to offer cutting-edge biodesign automation tools

GoInformatics and TeselaGen have announced a strategic partnership to offer a state-of-the-art Biological Design Automation and Scientific Data Management platform. With a secure repository to collect, organize, and store research information, the integrated cloud-based platform will enable scientists advance their research at a much faster pace.

16 Nov 2013

"One-two punch" disables tumors' defenses

An aggressive form of breast cancer known as "triple negative" is very difficult to treat: Chemotherapy can shrink such tumors for a while, but in many patients they grow back and gain resistance to the original drugs.

25 Oct 2013

Small interfering RNAs in mammals act as specific antiviral weapons

Many viral infections are nipped in the bud by the innate immune response. This involves specific proteins within the infected cell that recognize the virus and trigger a signalling cascade - the so-called interferon response. This activates a protective mechanism in neighbouring cells and often results in the death of the primarily infected cell.

11 Oct 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

Arrowhead initiates Phase 1 study of ARC-520 for treatment against chronic HBV infection

Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it completed enrollment in a Phase 1 clinical trial of ARC-520, its RNAi-based candidate against chronic hepatitis B virus infection.

9 Oct 2013

Cholesterol-lowering drugs could be alternative treatment for people who take statins

A new class of cholesterol-lowering drugs that target a recently discovered regulator of harmful cholesterol could be an alternative or complementary treatment for the 30 million people who take statins, after the first trial in humans confirms the technique’s feasibility and safety.

4 Oct 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

RXi Pharmaceuticals reports positive results from second Phase 1 study with RXI‑109

RXi Pharmaceuticals Corporation, a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted technologies, today announced positive results from their second placebo-controlled Phase 1 study with RXI‑109, an sd-rxRNA compound that targets Connective Tissue Growth Factor, and is being developed for the treatment of abnormal dermal scars such as hypertrophic scars and keloids in conjunction with scar revision surgery.

12 Jul 2013

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.

12 Jul 2013

New approach delivers RNA drugs to silence malfunctioning genes

Silencing genes that have malfunctioned is an important approach for treating diseases such as cancer and heart disease. One effective approach is to deliver drugs made from small molecules of ribonucleic acid, or RNA, which are used to inhibit gene expression. The drugs, in essence, mimic a natural process called RNA interference.

10 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013