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.
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.
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.
The absence of a protein called SMG1 could be a contributing factor in the development of Parkinson's disease and other related neurological disorders, according to a study led by the Translational Genomics Research Institute.
Some of the most dangerous cancers are those that can outmaneuver the very drugs designed to defeat them, but researchers are now reporting a new Trojan-horse approach. In a preliminary study in the journal ACS Nano focusing on a type of breast cancer that is highly resistant to current therapies, they describe a way to sneak small particles into tumor cells, lower their defenses and attack them with drugs, potentially making the therapy much more effective.
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.
Molecular Templates, Inc., a biopharmaceutical company focused on the discovery and development of a new class of biologic therapeutics, announced today that it recently secured an $8.5M series C financing led by Excel Venture Management. Santé Ventures, an existing investor, also participated in the round.
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).
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.
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.
Researchers at The Mount Sinai Medical Center have developed an innovative system to test how a virus interacts with cells in the body — to see, for example, what happens in lung cells when a deadly respiratory virus attacks them.
Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that its long term partner, Shire has extended the existing partnership in the rare genetic disease space.
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.
A new strategy that dismantles a viral genome in human lung cells will ensure safe research on deadly strains of influenza, say researchers from the Icahn School of Medicine at Mount Sinai.
Mitosis is the process by which a cell gives rise to two daughter cells. It’s the basis behind the growth of the human body, the renewal of the human body as it ages, and unfortunately, the growth of cancers.
ARMAGEN announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II).
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.
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.
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.
Nanoparticles that deliver short strands of RNA offer a way to treat cancer and other diseases by shutting off malfunctioning genes. Although this approach has shown some promise, scientists are still not sure exactly what happens to the nanoparticles once they get inside their target 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.
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