Strategy

Our vision is to transform peoples' lives around the world by silencing diseases through our precision engineered medicines and driving positive change for the communities around us. 
 

With decades of scientific, molecular design and industry know-how, we create medicines that harness the body’s natural mechanism of RNA interference, or RNAi, to precisely target and silence disease-associated genes expressed in the liver. We are leveraging a hybrid business model based on our proprietary mRNAi GOLD™ Platform to rapidly advance a growing pipeline of wholly owned programs and partnership programs focused in areas of high unmet need.

Our Approach

Our core expertise is in the design and development of powerful siRNA therapeutics both with respect to gene target specificity as well as potency and duration of knockdown. We design our siRNAs with patients in mind- we want to improve outcomes for people suffering from debilitating conditions by safely and specifically knocking down disease-causing genes. Our experienced Clinical, Regulatory and CMC teams are experts in the delivery of complex clinical programs, translating discoveries into medicines which can change patient’s lives.

Our Technology

Our siRNA platform is a modular technology composed of an siRNA molecule directly conjugated to a targeting ligand via a proprietary linker. In our mRNAi GOLDTM Platform we use a GalNAc sugar as targeting moiety which allows for specific delivery of siRNAs to liver hepatocytes. The mRNAi GOLDTM Platform is well validated, with multiple clinical studies ongoing, and broad strategic collaborations in place.