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In genetics, microRNAs (miRNA) are single-stranded RNA molecules of 21-23 nucleotides in length, which regulate gene expression. miRNAs are encoded by genes from whose DNA they are transcribed but miRNAs are not translated into protein (i.e. they are non-coding RNAs); instead each primary transcript (a pri-miRNA) is processed into a short stem-loop structure called a pre-miRNA and finally into a functional miRNA. Mature miRNA molecules are partially complementary to one or more messenger RNA (mRNA) molecules, and their main function is to down-regulate gene expression.

New LNP compositions that improve systemic delivery of RNAi therapeutics discovered

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today announced the publication of new data in the journal Nature Biotechnology by Alnylam scientists and collaborators from Tekmira Pharmaceuticals Corporation, AlCana Technologies, Inc., and The University of British Columbia (UBC). The new study employed a rational design approach for the discovery of novel lipids that can be incorporated into lipid nanoparticles (LNPs) for systemic delivery of RNAi therapeutics.

20 Jan 2010

Alnylam Pharmaceuticals presents data on multiple therapeutic programs at RNAi Symposium

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today announced that it presented data from multiple pre-clinical and clinical programs at the “RNA Silencing: Mechanism, Biology, and Application” Keystone Symposium held January 14-19, 2010 in Keystone, Colorado. Alnylam and its collaborators presented data from Alnylam’s therapeutic programs including transthyretin (TTR)-mediated amyloidosis and Huntington’s disease, as well as new data on delivery approaches for the systemic delivery of RNAi therapeutics.

20 Jan 2010

Regulus Therapeutics presents new data on oncology, immune disease and HCV therapeutic programs

Regulus Therapeutics Inc. today announced that new pre-clinical data from multiple therapeutic programs were presented at the “RNA Silencing: Mechanism, Biology and Application” Keystone Symposium held January 14 – 19, 2010 in Keystone, Colorado. Regulus and its collaborators presented data showing microRNA target regulation by anti-miRs, as well as data from therapeutic programs focused on oncology, immune disease and hepatitis C virus (HCV) infection.

18 Jan 2010

Researchers identify ways to predict patients with small-cell lung cancer

Researchers for TGen Clinical Research Services at Scottsdale Healthcare (TCRS) have identified a way to predict which patients with small-cell lung cancer may be resistant to first-line chemotherapy.

18 Jan 2010

miRagen Therapeutics establishes SAB

miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients’ lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, announced today the formation of the company’s world renowned Scientific Advisory Board (SAB). The SAB was established to provide miRagen with valuable guidance in developing an extensive pipeline of microRNA-based drugs and is comprised of leaders who are internationally recognized experts in their respective fields.

12 Jan 2010

Isis Pharmaceuticals adds ISIS-APOCIIIRx to its development pipeline to treat TG

Isis Pharmaceuticals, Inc. announced today that it has added a new drug, ISIS-APOCIIIRx, to its development pipeline. ISIS-APOCIIIRx provides a novel approach to treat hypertriglyceridemia, or high levels of triglycerides (TG).

12 Jan 2010

Mirna Therapeutics receives ETF award to develop miRNA therapeutics for multiple cancers

Mirna Therapeutics announced today that the Company received a $5 million award from the State of Texas through the Texas Emerging Technology Fund (“ETF”). The ETF was created with the goal of growing new and existing businesses to accelerate the introduction of new products and services in the marketplace. This is the largest award of this type that has ever been granted.

11 Jan 2010

Key mechanism for systemic delivery of RNAi therapeutics using LNPs discovered

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has discovered a key mechanism related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs). The new pre-clinical research was presented at the “Advances in Biopharmaceuticals” Keystone Symposium held January 8-13, 2010 in Midway, Utah, and was performed in collaboration with scientists at the Max Planck Institute of Molecular Cell Biology and Genetics.

9 Jan 2010

Alnylam Pharmaceuticals receives payment from Roche to initiate pre-IND studies for RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has received a milestone payment from its partner Roche related to initiation of pre-IND (investigational new drug application) studies for an RNAi therapeutics product candidate.

7 Jan 2010

Advances in development of “lipidoid” formulations for systemic delivery of RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators from the David H. Koch Institute for Integrative Research at the Massachusetts Institute of Technology (MIT) today announced the publication of new data in the journal Proceedings of the National Academy of Sciences (PNAS) describing further advancements in discovery and development of novel “lipidoid” formulations for the systemic delivery of RNAi therapeutics.

30 Dec 2009

Tel Aviv University's new Genome Analyzer fuels research dreams and tomorrow's cures

To identify the hemophilia mutation that affected Queen Victoria and her European relatives, scientific detectives used a cutting-edge "deep sequencing tool." Able to trace rare genetic disease mutations, the tool can turn a single laboratory into a fertile genetic research center.

22 Dec 2009

OncoGenex Pharmaceuticals to pay Isis Pharmaceuticals $10M for license of OGX-011 to Teva Pharmaceutical Industries

Isis Pharmaceuticals, Inc. today announced that it will receive a $10 million payment from OncoGenex Pharmaceuticals, Inc. as a result of OncoGenex' license of OGX-011 to Teva Pharmaceutical Industries Ltd. ( TEVA). OGX-011 is a second-generation antisense drug co-discovered by Isis and OncoGenex that has completed a successful Phase 2 program in patients with advanced prostate cancer and advanced non-small cell lung cancer.

21 Dec 2009

Rosetta Genomics announces third-quarter 2009 financial results

Rosetta Genomics, Ltd., a leading developer of microRNA-based molecular diagnostics, today reported financial results for the three and nine months ended September 30, 2009 and provided a business update.

18 Dec 2009

Regulus Therapeutics wins exclusive rights to modulate microRNAs

Regulus Therapeutics Inc., founded by Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc., announced today that it has obtained exclusive rights from the California Institute of Technology to inventions made in the laboratory of David Baltimore, Ph.D. covering methods and compositions for modulating microRNA-146 and microRNA-155.

18 Dec 2009

Human MicroRNA Panel for gene-expression profiling to be launched by WaferGen Biosystems

WaferGen Biosystems, Inc., a leading developer of state-of-the-art genetic analysis systems, today announced that it will be launching the Human MicroRNA Panel for its innovative service for gene-expression profiling using its SmartChip(TM) Real-Time PCR System.

17 Dec 2009

Scientists rediscover Cyclin G1 gene as prime target for anti-cancer therapies

Epeius Biotechnologies Corporation, an emerging leader in the field of targeted genetic medicine, gained international validation of the cutting-edge science behind its lead oncology product, Rexin-G, when scientists around the world rediscovered the Cyclin G1 gene to be a major locus of cancer pathogenesis and disease progression, and thus a prime target for anti-cancer therapies.

16 Dec 2009

USPTO issues Notice of Allowance for U.S. patent application in the 'Crooke' patent family

Isis Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. announced today that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. patent application (No. 10/078,949) in the “Crooke” patent family, which broadly covers chemically modified RNA-containing therapeutics.

16 Dec 2009

UT researchers identify new molecule that slows ALS progression in mice

Researchers at UT Southwestern Medical Center have found that a molecule produced naturally by muscles in response to nerve damage can reduce symptoms and prolong life in a mouse model of amyotrophic lateral sclerosis (ALS).

11 Dec 2009

microRNA-206 plays crucial role in the progression of ALS

miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients' lives by developing innovative microRNA-based therapeutics for cardiovascular and muscle disease, today announced the publication of data demonstrating that microRNA-206 (miR-206) plays a crucial role in the progression of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease) and in neuromuscular synaptic regeneration in mice.