RNAi Therapeutics News and Research

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RNA interference (RNAi) was discovered less than a decade ago and already there are human clinical trials in progress or planned. A major advantage of RNAi versus other anti-sense based approaches for therapeutic applications is that it utilizes cellular machinery that efficiently allows targeting of complementary transcripts, often resulting in highly potent down-regulation of gene expression.

Alnylam Pharmaceuticals presents data on RNAi therapeutic targeting PCSK9 for hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the presentation of new pre-clinical data from its hypercholesterolemia program, performed in collaboration with scientists at the University of Texas Southwestern Medical Center at Dallas. The data were presented at the PCSK9 Conference: From Gene to Therapeutics held in Nantes, France from March 11-13, 2010.

15 Mar 2010

Alnylam Pharmaceuticals asks Court to dismiss MIT from any claims for monetary damages in ongoing litigation

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the company and Max Planck have asked the Court to dismiss the Massachusetts Institute of Technology from any claims for monetary damages in the ongoing litigation related to the Tuschl I patent series.

4 Mar 2010

Alnylam Pharmaceuticals initiates ALN-RSV01 Phase IIb trial in adult lung transplant patients for RSV infection

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a Phase IIb trial in adult lung transplant patients with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection. RSV infection in lung transplant patients represents an important unmet medical need; the condition is associated with significant morbidity, including the development of acute lung transplant rejection in 10% to 20% of infected patients.

22 Feb 2010

Alnylam Pharmaceuticals receives 'Notice of Allowance' from USPTO

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has awarded a Notice of Allowance for the “Soutschek and Manoharan” patent (Application No. 10/916,185), which covers certain chemically modified siRNAs of any length directed to any target, including siRNAs with sterol conjugates for in vivo delivery.

9 Feb 2010

Silence Therapeutics strengthens patent portfolio of RNAi therapeutics in solid tumor treatment

Silence Therapeutics plc today announced that the United States Patent and Trademark Office (USPTO) has issued the company a notice of allowance on a patent application directed to methods of treatment using a high-value therapeutic target in the area of oncology. Known as PKN-3, the target is a protein kinase C-related molecule that is believed to play an important role in the growth of cancer cells, as well as metastasis formation.

8 Feb 2010

Access Pharmaceuticals initiates Cobalamin technology program for targeted delivery of siRNA therapies

ACCESS PHARMACEUTICALS, INC. announced today that it initiated an internal pre-licensing program to confirm the utility of its proprietary Cobalamin (vitamin B12) platform technology for targeted delivery of siRNA therapies. The program is considered important because, despite the widely publicized potential of RNA therapy, researchers up to now have been stymied in their efforts to design a pharmaceutical product that efficiently transports siRNA therapeutics into the cells they are designed to inhibit or kill.

5 Feb 2010

Global health community to be engaged in using resources of GSK and Alnylam IP Pool

BIO Ventures for Global Health, GlaxoSmithKline (GSK), and Alnylam announced today that the organizations are partnering to engage the global health community in using the powerful resources of the GSK and Alnylam Intellectual Property (IP) Pool.

21 Jan 2010

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

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

DSMB recommends Quark Pharmaceuticals to continue clinical study of QPI-1002

Quark Pharmaceuticals, Inc., the world leader in clinical development of RNAi-based therapeutics, announced today that the independent Data Safety Monitoring Board ("DSMB") recommended that QPI-1002 (I5NP) continue on to the next phase of clinical testing.

5 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

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

Silence Therapeutics and Intradigm merge to form RNAi

The Boards of Silence Therapeutics plc and Intradigm Corporation are delighted to announce the merger of their businesses to form a leading company in the field of RNAi (RNA interference).

17 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

Regulus Therapeutics to host educational webinar about microRNA biology

Regulus Therapeutics Inc., founded by Alnylam Pharmaceuticals, Inc., and Isis Pharmaceuticals, Inc., unveiled plans for an educational webinar about microRNA biology that will highlight several recent exciting discoveries in the field and showcase the therapeutic strategies based on microRNA.

10 Dec 2009

New pre-clinical data from Alnylam Pharmaceuticals' ALN-VSP program presented

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today announced new pre-clinical data from its ALN-VSP program presented at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics International conference being held November 15 - 19, 2009 in Boston, Mass.

18 Nov 2009

RXi Pharmaceuticals announces financial results for the quarter ended September 30, 2009

RXi Pharmaceuticals Corporation, a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today reported its financial results for the quarter ended September 30, 2009.

16 Nov 2009

Cequent Pharmaceuticals proposes the first test of an orally administered RNA interference drug in humans

Cequent Pharmaceuticals, a pioneer in the development of novel products to deliver RNAi-based treatments to prevent and treat human disease, has announced that it has filed its first IND (investigational new drug) application with the U.S. Food and Drug Administration (FDA).

12 Nov 2009