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Exosomes can transfer membrane proteins, study reveals

Important new research from UMass Medical School demonstrates how exosomes shuttle proteins from neurons to muscle cells where they take part in critical signaling mechanisms, an exciting discovery that means these tiny vehicles could one day be loaded with therapeutic agents, such as RNA interference, and directly target disease-carrying cells.

28 Mar 2013

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

19 Mar 2013

Insight into how Th17 cell growth influences development of immune responses

New work from the Broad Institute's Klarman Cell Observatory, Brigham and Women's Hospital, Harvard University, MIT, and Yale University expands the understanding of how one type of immune cell - known as a T helper 17 or Th17 cell - develops, and how its growth influences the development of immune responses.

7 Mar 2013

RNAi effective in treating cancer patients

The new study published in Cancer Discovery, the flagship journal of the American Association of Cancer Research (AACR), involving three Spanish and six American research centres, presents significant results in treating cancer patients with nanoparticles containing ribonucleic acid interference (RNAi) molecules.

13 Feb 2013

Researchers identify miR-506 as potential therapeutic candidate for metastatic ovarian cancer

Researchers employed an extensive analysis of genomic information to identify a new, high-risk cohort of ovarian cancer patients, characterize their tumors, find a potential treatment and test it in mouse models of the disease.

11 Feb 2013

SINEUP allows scientists to target individual genes in cells to increase protein production

One of the most innovative biotechnologies of the last decade has recently been developed. SINEUP allows scientists, for the first time, to target individual genes in cells to knockup, or increase, the amount of protein they make. The technique will improve Protein manufacture, analyse the function of genes and engineer improved cell function.

From Cell Guidance Systems 31 Jan 2013

Scientists design small molecules that recognize myotonic dystrophy-associated RNAs

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by designing molecules that recognize and disable RNA targets. As a proof of principle, in the new study the team designed a molecule that disabled the RNA causing myotonic dystrophy.

21 Dec 2012

Alnylam advances Tuschl II patent estate through the USPTO

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office has issued Notices of Allowance for three patent applications from the company's exclusively held Tuschl II patent estate.

26 Nov 2012

Study shows how MCUR1 protein helps regulate movement of calcium into mitochondria

A team of scientists from Temple University School of Medicine and the University of Pennsylvania has moved another step closer to solving a decades-long mystery of how the all-important flow of calcium into the cell's power source, the mitochondria, is controlled.

26 Nov 2012

ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, "The Liver Meeting") held November 9-13, 2012 in Boston.

15 Nov 2012

Alnylam third quarter 2012 revenues decrease to $16.8 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2012, and company highlights.

6 Nov 2012

Genzyme, Alnylam partner to develop and commercialize RNAi therapeutics against ATTR

Alnylam Pharmaceuticals, Inc. and Genzyme, a Sanofi company, announced today that they have formed an exclusive alliance to develop and commercialize RNAi therapeutics targeting transthyretin (TTR) for the treatment of transthyretin-mediated amyloidosis (ATTR) in Japan and other Asia-Pacific countries.

23 Oct 2012

RXi receives SBIR grant to enable discovery and preclinical development of sd-rxRNAs

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 that it was awarded a Small Business Innovation Research (SBIR) grant by the National Cancer Institute (NCI) of the National Institutes of Health (NIH).

5 Oct 2012

U.S. District Court rules in favor of Alnylam in patent infringement case against Tekmira

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the U.S. District Court of the District of Massachusetts has ruled in favor of Alnylam and has denied Tekmira Pharmaceuticals Corporation's motion to dismiss certain claims and motion to disqualify Alnylam's counsel in an infringement suit filed in January 2012.

26 Sep 2012

RXi completes dosing in RXI-109 Phase 1 study for dermal scarring

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 that dosing with their first drug in its first Phase 1 study, was completed last week.

24 Sep 2012

Recent data from Silence Therapeutics’ Atu111 pre-clinical study on acute lung injury

Silence Therapeutics plc, a leading RNA interference (RNAi) therapeutics company is pleased to announce that recent data in pre-clinical models demonstrated the efficacy of Atu111 in acute lung injury.

10 Sep 2012

Alnylam announces complete results from ALN-RSV01 Phase IIb trial on RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today complete results from its Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients.

6 Sep 2012

Enrollment complete in Arrowhead’s CALAA-01 phase 1b trial on solid tumors

Arrowhead Research Corporation, a targeted therapeutics company, today announced that, through its Calando Pharmaceuticals subsidiary, it has completed enrollment of a phase 1b clinical trial of CALAA-01, its RNAi therapeutic candidate targeting solid tumors. All patients participating in the trial have completed dosing with the exception of one patient who is still receiving treatment and will continue to be monitored.

31 Aug 2012

New results describe development of DC cancer vaccine with enhanced immunogenic potential

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators at Radboud University Nijmegen Medical Centre in the Netherlands announced today that they have published new pre-clinical results in the journal Cancer Immunology Immunotherapy describing the development of a dendritic cell (DC) cancer vaccine with enhanced immunogenic potential.

23 Aug 2012

RNA-delivering nanoparticles allow rapid screening of new drug targets in mice

By sequencing cancer-cell genomes, scientists have discovered vast numbers of genes that are mutated, deleted or copied in cancer cells. This treasure trove is a boon for researchers seeking new drug targets, but it is nearly impossible to test them all in a timely fashion.

16 Aug 2012