PBGENE-PMM

PBGENE-PMM is a first-in-class, in vivo gene editing therapeutic developed by Precision BioSciences for the treatment of m.3243-associated primary mitochondrial myopathy (PMM). It utilizes the proprietary ARCUS platform to deliver a mitochondria-targeted engineered meganuclease (mitoARCUS) that specifically recognizes and eliminates mutant mitochondrial DNA (mtDNA), particularly the m.3243A>G mutation. This approach allows wild-type mtDNA to repopulate the cell, restoring normal mitochondrial function and shifting heteroplasmy toward healthy mtDNA. Unlike CRISPR-based tools, mitoARCUS is a single-component protein capable of direct mitochondrial delivery without guide RNAs or nucleic acids. Preclinical data demonstrate robust elimination of mutant mtDNA with minimal off-target effects in nuclear DNA and significant improvements in cellular respiration and function[1][2][5]. The therapy is designed as a potential one-time curative treatment for PMM patients.

Brand names

PBGENE-PMM

Other names

PBGENE-3243PBGENE3243PBGENE 3243Mutant Mitochondrial DNA Elimination Program