Pipeline

Present and future focus

We are currently focused on developing treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. However, RNA therapeutics have the potential to address a vast array of genetic, protein-based diseases.

Program

Phase

Cystic Fibrosis

Preclinical

OTC

Preclinical

Rare Liver Disease

Discovery

Rare Lung Disease

Discovery

Areas of Impact

Changing the course of disease