Year: 2018-19

Company: Alnylam Pharmaceuticals

Liaison(s): Gautham Sridharan, Fred Trembley, Shiri Tsour, Lucas BonDurant

innovative medicines with the potential to improve the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made.

Alnylam and the TMP project team at the Keck Graduate institute (KGI) are working together to discover and evaluate new drug targets. Alnylam’s platform technology based on RNA interference enables the silencing of disease-causing proteins within the liver. Within this context, the team at KGI has performed a deep dive on the feasibility of targeting five liver-enriched genes that Alnylam provided for cardio-metabolic disease phenotypes. In addition, the team has proposed a novel artificial intelligence-based approach to discovering new targets for disease using natural language processing (NLP). Their work has contributed to Alnylam’s decision to conduct in vivo experiments for further validation of targets.