PBGENE-PH1

PBGENE-PH1 is an in vivo gene-editing therapeutic candidate from Precision BioSciences designed to treat primary hyperoxaluria type 1 by knocking out the hepatic HAO1 gene (encoding glycolate oxidase) using the ARCUS meganuclease platform, with the goal of reducing oxalate production as a one-time treatment. Preclinical programs reported robust HAO1 protein knockdown in non-human primates after a single administration using AAV delivery, and Precision later pursued lipid nanoparticle mRNA delivery, with IND-enabling activities initiated and an IND/CTA targeted in 2023. PH1 is a rare genetic liver-origin metabolic disorder leading to oxalate overproduction, kidney stones, nephrocalcinosis, and kidney failure.[4][6][5][7]