Reimagining RNA medicine

Our RNA medicines platform, PRISM^®, combines multiple modalities, chemistry innovation, and deep insights in human genetics to deliver scientific breakthroughs that treat both rare and prevalent disorders. Our toolkit of RNA-targeting modalities includes editing, splicing, RNA interference and antisense silencing, providing us with unmatched capabilities for designing and sustainably delivering candidates that optimally address disease biology.

Our diversified pipeline includes clinical programs in obesity, alpha-1 antitrypsin deficiency (“AATD”), Duchenne muscular dystrophy (“DMD”), and Huntington’s disease (“HD”), as well as several preclinical programs utilizing our versatile RNA medicines platform. Driven by the calling to “Reimagine Possible,” we are leading the charge toward a world in which human potential is no longer hindered by the burdens of disease.