– Company Expects to Initiate a Phase 1/2 Study for ALN-KHK in Early 2023, with Initial Results Expected in Late 2023 – “We are excited to advance ALN-KHK to the clinic as our first RNAi therapeutic ...

RNA interference (RNAi) offers a fast and easy system to study gene functions. However, the traditionally used small interfering RNAs (siRNAs) can exhibit pronounced and problematic off-target effects ...

RNA interference (RNAi) is an ancient genetic regulatory mechanism that modern-day scientists are trying to harness. Since its discovery more than 10 years ago, RNAi R&D has run the gamut from ...

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 ...

A Cornucopia of siRNA- and RNAi-based Therapeutics in Development Show Promise Researchers from academia and industry will present the latest developments in the application of interference RNA (RNAi) ...

- Company Expects to Initiate a Phase 1 Study in Early-Onset Alzheimer’s Disease in Early 2022, with Initial Human Data at or Around Year-End 2022 - - ALN-APP Marks the Industry’s First-Ever RNAi ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced the submission of a Type II Variation to the ...

Company’s first clinical-stage program headlines a diverse and novel pipeline poised to deliver 1-2 new investigational drugs into the clinic annually CAMBRIDGE, Mass., November 10, 2025--(BUSINESS ...

Ribonucleic acid (RNA) interference is a relatively new technique in which small molecules called short interfering RNA (siRNA) can be inserted into cells to turn off a chosen gene. The potential uses ...

This article explores how challenges such as sequence optimization, immune activation and off-target effects are being addressed to enhance the safety, durability and efficacy of RNA therapeutics.