dicerna

Dicerna Pharmaceuticals, commonly referred to as Dicerna, is a biotechnology company that develops RNA interference (RNAi) therapeutics. The company focused its work on exploiting the RNAi pathway to silence disease-causing genes, with an emphasis on liver-expressed targets and systemic delivery. Central to Dicerna’s approach is a proprietary platform intended to improve delivery of small interfering RNA (siRNA) to hepatocytes and other cells.

Technology and approach: RNA interference is a cellular mechanism whereby siRNA molecules guide the RNA-induced silencing complex (RISC) to complementary messenger RNA, triggering degradation and lowering the production of the target protein. Dicerna advanced a platform that aimed to enhance siRNA stability, specificity, and tissue delivery, particularly to the liver. The platform leveraged chemical modifications and targeting strategies to facilitate uptake by liver cells, enabling systemic administration of RNAi agents.

Pipeline and development: Dicerna developed multiple discovery and development programs targeting genes implicated in hepatic and metabolic diseases, as well as other genetic disorders. The company pursued collaborations with other pharmaceutical and biotechnology companies to advance its RNAi programs, reflecting a broader industry effort to translate RNA interference technologies into therapeutics.

History and status: Founded in the mid-2000s, Dicerna contributed to the field of RNAi therapeutics through its GalXC-inspired strategies for liver-targeted gene silencing and collaboration-driven development. The company’s work situates it within the broader evolution of RNAi medicine, alongside other teams pursuing siRNA delivery and tissue targeting.