Companies in #Duchenne muscular dystrophy
Found 1 company operating in this sector.
Precision BioSciences (NASDAQ: DTIL) is a Durham, North Carolina-based clinical-stage gene editing company built on ARCUS, a proprietary genome editing platform derived from a naturally occurring homing endonuclease (I-CreI) that is small enough for viral and non-viral delivery and is designed to cut, excise and insert DNA at highly specific sites. The company's wholly owned pipeline is focused on in vivo editing: PBGENE-HBV for chronic hepatitis B, designed to eliminate covalently closed circular DNA (cccDNA) with repeat lipid nanoparticle dosing, and PBGENE-DMD for Duchenne muscular dystrophy, designed to restore near full-length dystrophin for patients with mutations between exons 45 and 55. Earlier programmes include PBGENE-3243 for m.3243-associated mitochondrial disease and PBGENE-PMM for primary hyperoxaluria, alongside the partnered iECURE-OTC programme in ornithine transcarbamylase deficiency. Precision previously licensed its allogeneic CAR T platform to Imugene in 2023 and has been funded through a series of public offerings and institutional placements.