Value proposition

Unlocking the broad potential of RNA medicines to transform human health

We are a biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health.

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 common 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 Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy, Huntington’s disease, and obesity, as well as several preclinical programs utilizing our broad RNA therapeutics toolkit.

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 burden of disease.

Biotechnology, Oligonucleotides, Genetic medicines, RNA editing, RNAi, Duchenne muscular dystrophy, Alpha-1 antitrypsin deficiency, Huntington's Disease, and Obesity