Precision BioSciences Presents Preclinical Data Highlighting PBGENE-PMM as a Potential Therapy for Primary Mitochondrial Myopathy
These data are being presented at the Mitochondrial Medicine – Therapeutic Development Annual Conference, being held from March 18-20, 2024 in Hinxton, UK.
- These data are being presented at the Mitochondrial Medicine – Therapeutic Development Annual Conference, being held from March 18-20, 2024 in Hinxton, UK.
- “Today’s data from our PBGENE-PMM program further validate our work with ARCUS and continue to demonstrate its ability to make precise edits while avoiding off-target activity,” said Jeff Smith, PhD, Chief Research Officer of Precision BioSciences.
- PBGENE-PMM is designed to eliminate mutant mitochondrial DNA leaving normal functioning wild-type mitochondrial DNA intact to repopulate the cell, resulting in a shift in heteroplasmy and improvement in mitochondrial function.”
Smith continued, “Additionally, ARCUS can cross mitochondrial membranes in order to access the mitochondrial DNA. - Together, these data support the development of PBGENE-PMM as a single-treatment, in vivo gene editing therapeutic for m.3243-associated primary mitochondrial myopathy.