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, ...

LEXINGTON, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ...

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 ...

Gift Article 10 Remaining As a subscriber, you have 10 articles to gift each month. Gifting allows recipients to access the article for free. AbbVie is entering a collaboration and license option deal ...

When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury. But in neurological disorders such as ALS and spinal muscular atrophy, or following spinal ...