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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

Researchers provide new revelations about intricate pathways involved in turning on T cells

A major study from researchers at the La Jolla Institute for Allergy and Immunology provides new revelations about the intricate pathways involved in turning on T cells, the body's most important disease-fighting cells, and was published today in the prestigious scientific journal Nature.

24 Jun 2013

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.

21 Jun 2013

Human Argonautes directly cuts messenger RNA

Human Argonautes (hAgo), are key proteins involved in a process known as RNA interference. RNAi, as it is often called, is a mechanism that cells use to regulate gene expression.

7 Jun 2013

Arcturus Therapeutics closes $1.3 M in seed funding round

Arcturus Therapeutics, Inc., an industry leader in RNAi technologies for the treatment of disease, today announced it has raised $1.3 million in a seed funding round led by multiple high net-worth private investors from the United States and Canada.

3 Jun 2013

Cenix BioScience, Debiopharm partner to develop novel therapeutic drug candidates

Cenix BioScience, a leading preclinical contract research provider and technology developer specialized in RNAi-, miRNA- and high content-driven pharmacology, and Debiopharm Group, a Swiss-based global biopharmaceutical group of companies with a focus on the development of prescription drugs that target unmet medical needs, including oncology and companion diagnostics, today announced that they have signed a research agreement to support Debiopharm in its ongoing efforts to develop novel therapeutic drug candidates.

23 May 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

Tacere Therapeutics submits TT-034 clinical trial application to Recombinant DNA Advisory Committee

RNAi-based therapeutics company Benitec Biopharma Limited today announced that the company's wholly owned US subsidiary, Tacere Therapeutics Inc., has submitted an application to the US National Institutes of Health's Recombinant DNA Advisory Committee.

18 Apr 2013

Benitec to conduct phase I/II clinical trial of TT-034 in patients with HCV in UCSD site

RNAi-based therapeutics company Benitec Biopharma Limited today announced the selection of the University of California, San Diego, Health Sciences as the second site for its upcoming phase I/II first-in-man trial for TT-034 in Hepatitis C infections.

16 Apr 2013