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 Pharmaceuticals publishes ALN-RSV01 Phase IIa study results

Alnylam Pharmaceuticals, Inc. a leading RNAi therapeutics company announced today that it has published results in the American Journal of Respiratory and Critical Care Medicine from its double-blind, placebo-controlled, randomized Phase IIa study of ALN-RSV01 in lung transplant patients naturally infected with respiratory syncytial virus (RSV).

30 Sep 2010

Pfizer enters agreement to acquire FoldRx Pharmaceuticals

Pfizer Inc. and FoldRx Pharmaceuticals, Inc., a privately held drug discovery and clinical development company, today announced that they have entered into an agreement under which Pfizer will acquire FoldRx.

2 Sep 2010

Alnylam Pharmaceuticals to present results on RNAi therapeutics at ACS National Meeting

Alnylam Pharmaceuticals, Inc.,a leading RNAi therapeutics company, announced today that it will make a total of 16 oral presentations at the American Chemical Society (ACS) Fall 2010 240th National Meeting & Exposition being held in Boston, Mass. from August 22-26, 2010.

24 Aug 2010

Alpha-synuclein gene silencing effective for treating neurodegenerative disorders: Research

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators at The Parkinson's Institute and the Mayo Clinic have published new research findings in the journal Public Library of Science (PLoS). The new data show effective silencing of the alpha-synuclein gene with an RNAi therapeutic administered directly to the substantia nigra in the CNS of non-human primates.

24 Aug 2010

GM-CSF protein reduces AD pathology, reverses memory loss: Study

A signaling protein released during rheumatoid arthritis dramatically reduced Alzheimer's disease pathology and reversed the memory impairment of mice bred to develop symptoms of the neurodegenerative disease, a new study by the University of South Florida reports. Researchers found that the protein, GM-CSF, likely stimulates the body's natural scavenger cells to attack and remove Alzheimer's amyloid deposits in the brain.

23 Aug 2010

MMV joins Pool for Open Innovation against neglected tropical diseases

The Medicines for Malaria Venture (MMV) today became the first product development partnership (PDP) to contribute intellectual property to the Pool for Open Innovation against Neglected Tropical Diseases. MMV joins GlaxoSmithKline (GSK), Alnylam Pharmaceuticals, and the Massachusetts Institute of Technology (MIT) in contributing to the Pool, which is administered by BIO Ventures for Global Health (BVGH).

17 Aug 2010

Alnylam Pharmaceuticals reports significant progress in discovery of novel technologies for systemic delivery of RNAi therapeutics

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, and collaborators from the Massachusetts Institute of Technology (MIT), The University of British Columbia (UBC), AlCana Technologies, Inc., and Tekmira Pharmaceuticals Corporation, presented results from multiple ongoing research efforts regarding the discovery of novel technologies for the systemic delivery of RNAi therapeutics.

10 Aug 2010

Alnylam publishes research findings of Sort1 gene role in cardiovascular disease development

Alnylam Pharmaceuticals, Inc. a leading RNAi therapeutics company, announced today the publication of new research findings in the journal Nature describing the discovery and validation of the role of the gene Sort1 in the development of cardiovascular disease, including myocardial infarction (MI). This work was done with collaborators at University of Pennsylvania (UPenn) School of Medicine, Massachusetts General Hospital (MGH), and the Broad Institute. The collaborative effort combined genome-wide association studies (GWAS) and RNAi technology to identify and validate novel genes as targets for new therapies for heart disease.

6 Aug 2010

Alnylam second-quarter revenues increase to $26.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter ended June 30, 2010, and company highlights.

5 Aug 2010

Taoist Tai Chi internal arts of health celebrates 40 years

Dimitri Granovski, a Russian immigrant to Canada, says that after 20 years of chronic migraines, he's become painkiller-free by practising these arts for only three months. Samantha Albert of Stratford, Ontario, says they help her deal with the effects of a critical blood disorder called amyloidosis. Helen Gaunt a wine producer in Margaret River, Australia, attributes her smooth recovery from thyroid and breast cancer to their practice.

31 Jul 2010

FDA issues formal response to Nephros' 510(k) application for hemodiafiltration system

Nephros, Inc., a medical device company developing and marketing filtration products for therapeutic applications, infection control, and water purification, today reported that the U.S. Food and Drug Administration has issued a formal response to the company's 510(k) application.

8 Jul 2010

Alnylam Pharmaceuticals initiates dosing in Phase I human clinical study with ALN-TTR01

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today it has initiated dosing in a Phase I human clinical study with ALN-TTR01. The study is aimed at evaluating the safety and tolerability of ALN-TTR01 in patients with transthyretin -mediated amyloidosis, and is also designed to provide preliminary data on human proof of concept based on measurements of TTR serum levels.

7 Jul 2010

Alnylam receives Portuguese, Swedish, British approvals to initiate ALN-TTR01 Phase I study in ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that its applications for ALN-TTR01 have been given clearance by Portuguese, Swedish, and British regulatory authorities to begin clinical testing. The trial will begin enrolling patients shortly in a blinded, randomized, placebo-controlled, multicenter Phase I study.

29 Jun 2010

Alnylam presents data on novel delivery lipids at IBC 6th Annual Cell Line Development and Engineering conference

Alnylam Biotherapeutics, a division of Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the presentation of new data at the IBC 6th Annual Cell Line Development and Engineering conference held in San Francisco, California from June 21 - 24, 2010.

24 Jun 2010

Update on Regulus and sanofi-aventis collaboration

Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. announced today that, as a result of the newly formed alliance Regulus has established with sanofi-aventis, each company will receive a payment of $1.8 million from Regulus. This represents 7.5% of the $25 million upfront payment from sanofi-aventis to Regulus.

23 Jun 2010

Switching to healthier diet can slow or reverse cognitive impairment in patients with Alzheimer's Disease: Study

Patients in the early to moderate stages of Alzheimer's Disease could have their cognitive impairment slowed or even reversed by switching to a healthier diet, according to researchers at Temple University.

9 Jun 2010

Millennium announces data on VELCADE in treatment of patients with amyloidosis

Millennium: The Takeda Oncology Company today announced data examining the use of VELCADE in the treatment of patients with relapsed or refractory light chain (AL) amyloidosis. Amyloidosis is a rare condition in which excessive proteins build up in organs or tissues, such as the heart, kidney or liver. These data were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) held in Chicago, Illinois, June 4-8, 2010.

5 Jun 2010

Key mechanisms for delivery of LNP-encapsulated siRNAs to the liver

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the publication of pre-clinical research in the journal Molecular Therapy revealing key mechanisms related to the systemic delivery of RNAi therapeutics using lipid nanoparticles (LNPs).

12 May 2010

Mutation causing light chain amyloidosis identified

Light chain amyloidosis, a deadly protein misfolding disease, is caused by multiple mutations in cells that are intended to protect the body. Instead, the mutations send misfolded bundles of proteins through the bloodstream, potentially destroying the heart, kidneys, liver or other organs. Mayo Clinic researchers have identified one of these mutations and have shown that the molecule's shifting position is as important as its unique shape. The findings appear in the current issue of the journal Structure.