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.

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

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

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

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

Progress with GSK in advancing VaxiRNA technology earns Alnylam $3.2M milestone payment

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has earned a development milestone payment from GlaxoSmithKline, totaling $3.2 million. The milestone payment is related to progress in the companies' collaboration on the use of VaxiRNA technology for the production of GSK influenza vaccine.

30 Jul 2012

Positive results from Alnylam’s ALN-TTR02 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the achievement of positive clinical results from its Phase I trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis.

16 Jul 2012

New paper documents discovery of novel lipids, including MC3

Alnylam Pharmaceuticals, a leading RNAi therapeutics company, and collaborators announced today the publication of a scientific paper documenting the discovery of novel lipids used in second generation lipid nanoparticles (LNPs) for systemic delivery of RNAi therapeutics.

14 Jul 2012

FDA provides Orphan Drug Designation to Alnylam’s ALN-TTR02

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).

20 Jun 2012

Alnylam announces results from ALN-VSP Phase I study on liver cancers

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced results today from its Phase I extension study with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement.

5 Jun 2012

Top-line results from Alnylam’s ALN-RSV01 Phase IIb trial on RSV infection

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

31 May 2012

Final results from Alnylam’s ALN-TTR01 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today final results from its completed Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

11 May 2012

Alnylam first quarter revenues decrease to $20.6 million

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

4 May 2012

Positive results from Alnylam’s ALN-PCS Phase I trial on severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive results from its Phase I clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia.

21 Apr 2012

Alnylam commences dosing in ALN-TTR02 Phase I clinical trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The study is aimed at evaluating the safety and tolerability of ALN-TTR02 in healthy volunteers.

19 Mar 2012

Arrowhead, Axolabs enter into RNAi therapeutics strategic agreement

Arrowhead Research Corporation, a nanomedicine company with development programs in RNAi therapeutics and obesity, and Axolabs GmbH, a custom research organization offering preclinical solutions and consultancy in the field of oligonucleotide therapeutics, today announced that they have entered into a Strategic Alliance and Master Services Agreement.

5 Mar 2012

Alnylam commences underwritten public offering of 7,000,000 shares of common stock

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today announced that it has commenced an underwritten public offering of 7,000,000 shares of its common stock. All of these shares are being sold by Alnylam.

14 Feb 2012

New cERD method can detect RNAi activity in biological fluids

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that its scientists have discovered a novel method for monitoring RNAi activity in blood samples, representing a potential major advance for future development of RNAi therapeutics, other therapeutic modalities, and diagnostics.

10 Feb 2012

Alnylam receives U.S. patent allowance for LNP platform

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for patent application number 12/813,448, covering the company's proprietary second generation lipid nanoparticle (LNP) platform, including the "MC3" lipid.

30 Jan 2012

Alnylam sues Tekmira over LNP-formulated siRNA molecules

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has filed a patent infringement lawsuit against Tekmira Pharmaceuticals Corporation in the U.S. District Court of the District of Massachusetts.

18 Jan 2012