Raising the bar with next-generation science.

Based on our leadership and scientific expertise in nucleic acid therapeutics, we’ve developed a third-generation antisense platform using gene silencing oligonucleotides (GSOs). The third-generation antisense (3GA) technology is designed to silence harmful genes through short, unique complementary oligonucleotides that bind to DNA and, in effect, turn functional protein-producing DNA off.

We believe that, based on their validation in pre-clinical models, our gene silencing approach can overcome the current hurdles of antisense technologies, including efficient delivery without a carrier, reduced immunotoxicity, and increased potency for patients. We are evaluating opportunities to clinically demonstrate the advantages of our third-generation antisense platform in established disease targets in both oncology and rare diseases, as well as prioritizing novel targets for development and commercialization as well as partnering and outlicensing opportunities . As a result of these efforts, to date we have created 22 specific 3GA compounds to specific gene targets across a wide variety of therapeutic areas such as rare diseases, oncology, autoimmune disorders, metabolic conditions, single-point mutations and beyond.  We have selected our first target for clinical development in an undisclosed, however well-established liver target with validated animal models and clinical endpoints and applicability for both rare and broader disease application.  Our selection of this target will enable us to establish human proof of concept for the 3GA platform in 2018 as well as differentiate from all other RNA-based therapeutic approaches.  We also currently have a licensing agreement with GSK for an undisclosed renal target with a current goal of clinical candidate selection in the first quarter of 2018.