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 ...

-- Novel approach to gene knockdown uses proprietary CASi (Conditionally Activated siRNA) molecules optimized to “switch” on, activating siRNA therapeutics only in selected cells -- -- CASi platform, ...

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 ...

Alnylam Pharmaceuticals is a leader in RNAi therapeutics, with multiple FDA-approved treatments and a robust pipeline targeting rare and severe conditions. The company has strong growth potential, ...

Ribonucleic acid, also called RNA, is a molecule present in all living cells. It plays a critical role in transmitting genetic instructions from DNA and creating proteins. With the power to execute a ...