17:48 , Mar 28, 2019 |  BC Innovations  |  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...
21:22 , Jan 31, 2019 |  BC Innovations  |  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...
01:14 , Sep 30, 2017 |  BioCentury  |  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...
07:00 , Oct 3, 2016 |  BC Week In Review  |  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...
07:00 , Sep 29, 2016 |  BC Extra  |  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...