Using RNA to interfere with a gene’s expression of disease-causing proteins is a validated therapeutic approach, but so far, the products in this drug class only address liver proteins. Reducing ...

HELIOS-B is a global, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of AMVUTTRA (R) in adult patients with wtATTR or hATTR amyloidosis with ...

It was not until 2018 that the US FDA approved the first therapy developed using the technique. Since then, there’s been a steady, but slow, number of RNAi treatments coming into the market. The ROBO ...

With demand for its RNA interference meds booming, Alnylam Pharmaceuticals has commenced work to soup up its flagship ...

Through its innovative asymmetric siRNA platforms, OliX Pharmaceuticals is opening up huge opportunities in RNA interference-therapeutic development to address unmet needs for various indications ...

In drug discovery, very little of what’s learned from one project can typically be carried over to the next. “For every new target you go after with a small molecule, you basically start from scratch, ...

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the planned expansion of its state-of-the-art manufacturing facility in Norton, Massachusetts. The ...

RNA interference (RNAi) therapeutics have garnered significant attention in clinical research due to their potential for treating various diseases, including genetic disorders, viral infections and ...

With six drugs approved for commercial development, Cambridge-based drugmaker Alnylam Pharmaceuticals Inc. is investing $250 ...

- Company hosting an obesity/metabolic R&D webinar today, August 14, 2024, at 2:00 pm ET as Part IV of the 2024 Summer Series of R&D Webinars PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead ...

PHILADELPHIA, PA—Zilebesiran (Alnylam), an investigational therapeutic agent that interferes with messenger RNA to stop the production of angiotensinogen (AGT), lowers systolic blood pressure levels ...

Alnylam Canada ULC is pleased to announce that Health Canada has issued a Notice of Compliance (NOC) authorizing AMVUTTRA® (vutrisiran) for the treatment of cardiomyopathy in adult patients with ...