BioCentury | Apr 7, 2020
Product Development

April 6 Quick Takes: Second anemia approval for BMS, Acceleron therapy; plus EU approves trio of metabolic disease drugs, Dicerna-Alnylam and Axsome

Reblozyl gets second approval  FDA approved Reblozyl luspatercept-aamt from Bristol Myers Squibb Co. (NYSE:BMY) and Acceleron Pharma Inc. (NASDAQ:XLRN) to treat anemia in certain patients with myelodysplastic syndromes. It is the second indication for the...
BC Extra | Nov 22, 2019
Company News

Alnylam outlines ambitions, pipeline progress at R&D day

In its R&D day presentation Friday, Alnylam outlined its goals of becoming a self-sustaining company with at least eight approved drugs and 10 late-stage clinical candidates by 2025, with safer GalNAc conjugates, a late-stage amyloidosis...
BC Innovations | Mar 28, 2019
Translation in Brief

Tuning metabolism in liver organoids

An Alnylam-MIT collaboration has used RNAi technology to tune enzyme expression in hepatocyte-based organoids to better model drug metabolism during tox screens. The collaboration with Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) built on a series of papers...
BC Innovations | Jan 31, 2019
Emerging Company Profile

Apic: Dual-gene therapies

Apic Bio Inc.'s APB-101 is a dual-function gene therapy for AAT deficiency that silences the mutant AAT gene and augments wild-type AAT expression, which the company believes gives it greater efficacy and lower liver toxicity...
BioCentury | Sep 30, 2017
Product Development

Running interference

Editor’s Note: An earlier version of this story was published in BioCentury on Sept. 25. It has been updated to include reporting about safety and efficacy data that was not included in the original. The...
BC Week In Review | Oct 3, 2016
Clinical News

ALN-AAT: Development discontinued

Alnylam will discontinue development of ALN-AAT after interim data from a single-blind, U.K. Phase I/II trial in 26 healthy volunteers showed that single subcutaneous doses of ALN-AAT led to transient, asymptomatic and dose-dependent elevations in...
BC Extra | Sep 29, 2016
Clinical News

Alnylam switching to backup AAT molecule after safety signal

Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said it will discontinue development of ALN-AAT for alpha-1 antitrypsin (AAT; A1AT ; SERPINA1 ) deficiency-associated liver disease due to a low incidence of elevated liver enzymes observed in a Phase I/II...
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