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 initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in development for the treatment of TTR-mediated amyloidosis.

17 Dec 2013

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans.

10 Dec 2013

Takeda presents clinical study results of oral proteasome inhibitor MLN9708 in multiple myeloma patients

Takeda Pharmaceutical Company Limited today announced final Phase 1 and preliminary Phase 2 results of a study combining oral investigational MLN9708 administered twice a week with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma.

10 Dec 2013

BUSM & BMC win GCE grant to develop next-generation condom using nanotechnology

The department of radiology at Boston University School of Medicine (BUSM) and Boston Medical Center (BMC) is a Grand Challenges Explorations (GCE) winner, an initiative funded by the Bill & Melinda Gates Foundation.

26 Nov 2013

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

GSK Adds Another Program To Isis Antisense Collab

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7 Oct 2013

Domino transplant saves patient suffering from liver cancer

Tiffany Schwantes was going to die without a new liver. The 31-year old mother of two suffered from an aggressive form of liver cancer and, because of organ allocation rules, she could not be placed high enough on the liver transplant list to receive a new liver in a timely manner.

19 Sep 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

BMC, BUSM and JIPMER to jointly research on tuberculosis

Boston Medical Center and Boston University School of Medicine are partnering with the Jawaharlal Institute of Postgraduate Medical Education and Research to study tuberculosis. This research is supported by a five-year, $2.5 million grant from the Indo-U.S. Vaccine Action Program.

2 Aug 2013

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.

12 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.

21 Jun 2013

Takeda begins MLN9708 phase 3 clinical trial in newly diagnosed myeloma patients

Takeda Pharmaceutical Company Limited today announced the initiation of an international phase 3 clinical trial evaluating once a week MLN9708 in combination with lenalidomide and dexamethasone in patients with newly diagnosed multiple myeloma who are not candidates for transplant.

24 May 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

Alnylam begins dosing in Phase I clinical trial with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

19 Mar 2013

PET with 11C-PIB can positively visualize amyloid deposits in the heart

While amyloid imaging may now be most associated with detecting plaques in the brain, it has the potential to change the way cardiac amyloidosis is diagnosed. According to first-of-its-kind research published in the February issue of The Journal of Nuclear Medicine, positron emission tomography (PET) with 11C-PIB can positively visualize amyloid deposits in the heart.

5 Feb 2013

Amyloid clue to Parkinson’s dementia

Study findings link amyloid β burden to future cognitive decline in patients with Parkinson’s disease.

22 Jan 2013

Alnylam advances Tuschl II patent estate through the USPTO

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office has issued Notices of Allowance for three patent applications from the company's exclusively held Tuschl II patent estate.

26 Nov 2012

ALN RNAi therapeutic program represents novel approach for treatment of AAT deficiency

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of liver disease associated with AAT deficiency. These data were presented at the 63rd Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, "The Liver Meeting") held November 9-13, 2012 in Boston.

15 Nov 2012

Gene silencing technology used to fight RSV and influenza

New hope has arrived for the fight against two deadly viral infections, RSV and influenza, as Griffith researchers prepare to develop a cutting-edge approach to treatment delivery.

6 Nov 2012