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 levels of certain proteins in the central nervous system (CNS) could offer a new way to treat neurological disorders. The challenge facing drug developers is getting these therapies deep into the brain. Atalanta Therapeutics has technology that delivers RNA interference therapies into the CNS. The startup has spent the past four years quietly working on its technology and potential drugs. On Tuesday, Atalanta revealed two lead programs for rare neurological disorders that currently have no FDA-approved therapies. The Boston-based startup also announced $97 million in financing to advance those programs to the clinic and support other CNS therapies in its pipeline. An RNA interference (RNAi) therapy uses small pieces of non-coding RNA to block how certain genes are expressed. In 2018, Alnylam Pharmaceuticals’ Onpattro became the first FDA-approved RNAi drug. This therapy is delivered inside a lipid nanoparticle, which preferentially goes to liver cells. Alnylam and others have been pursuing ways to bring RNAi to the CNS.

Full report : Atalanta Therapeutics unveils $97 million in financing to fight two neuro diseases through RNA technology.