We are a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.
We aspire to develop best-in-class medicines across multiple therapeutic modalities using PRISM TM, our proprietary discovery and drug development platform that enables the precise design, optimization and production of stereopure oligonucleotides. Driven by a resolute sense of urgency, we are targeting a broad range of genetically defined diseases so that patients and families may realize a brighter future.
Foundation of Neurology Programs
Clinical Development Expertise