Amyloidosis News and Research

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Amyloidosis is the term applied to a group of conditions caused by deposits of an abnormal protein called amyloid. This rare but serious condition affects between 1 and 5 people in every 100,000 each year in the UK. The condition is mainly seen among the elderly.

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

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

Limited positive effect of Tafamidis in neurological disorders

Tafamidis meglumine was approved in November 2011 for the treatment of transthyretin amyloidosis in adults.

17 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

BUSM part of Transthyretin Amyloidosis Cardiac Study

Researchers at Boston University School of Medicine (BUSM) have been part of a multicenter observational study called TRACS (Transthyretin Amyloidosis Cardiac Study) to help determine the health significance of a particular gene mutation which is commonly found in Black Americans.

14 Aug 2012

Hint of tafamidis benefit in rare polyneuropathy

The results of a randomized trial suggest that tafamidis treatment may slow the progression of early-stage V30M transthyretin familial amyloid polyneuropathy.

2 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

BWH performs New England's first implant of SynCardia temporary Total Artificial Heart

SynCardia Systems, Inc., manufacturer of the world's first and only FDA, Health Canada and CE (Europe) approved Total Artificial Heart, announced today that Harvard-affiliated Brigham and Women's Hospital in Boston has performed New England's first implant of the SynCardia temporary Total Artificial Heart.

11 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

Pfizer receives FDA Complete Response Letter for tafamidis meglumine NDA

Pfizer Inc. announced today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) on its New Drug Application (NDA) for tafamidis meglumine.

19 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

Study shows how tafamidis drug works against amyloid disease

Scientists from The Scripps Research Institute and Pfizer Inc. have published a new study showing how a new drug called tafamidis (Vyndaqel) works.

31 May 2012

FDA advisory committee votes in favor of Pfizer's tafamidis meglumine

Pfizer Inc. announced today that the United States Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee voted on Pfizer's clinical data package for tafamidis meglumine.

25 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