DURHAM, N.C. - Precision BioSciences, Inc. (NASDAQ: NASDAQ:DTIL), a gene editing company, announced at the Mitochondrial Medicine Therapeutic Development Annual Conference in Hinxton, UK, significant progress in its development of a treatment for primary mitochondrial myopathy (PMM), a hereditary metabolic disorder.
The company's ARCUS genome editing platform has demonstrated the ability to selectively eliminate mutant mitochondrial DNA (mtDNA) associated with PMM without affecting nuclear DNA.
The data presented on Tuesday showed that Precision's gene editing candidate, PBGENE-PMM, could shift heteroplasmy by targeting and removing the mutant m.3243 mtDNA, allowing healthy wild-type mtDNA to proliferate and enhance mitochondrial function in cells.
This approach aims to provide a one-time, in vivo gene editing therapy for PMM, which currently has no curative treatments. Precision BioSciences anticipates submitting a Clinical Trial Application (CTA) and/or Investigational New Drug (IND) application for PBGENE-PMM in 2025.
Jeff Smith, PhD, Chief Research Officer at Precision BioSciences, highlighted the specificity of ARCUS for editing point mutations such as m.3243, noting its potential for mitochondrial DNA editing due to its single-protein nature that negates the need for guide-RNA. This contrasts with CRISPR-based editors, which cannot penetrate mitochondrial membranes.
The preclinical findings revealed that PBGENE-PMM-treated cells had a significant reduction in mutant mtDNA, with a corresponding increase in cellular respiration rates, indicating improved mitochondrial function. These results support further development of PBGENE-PMM as a potential single-treatment gene therapy for PMM.
ARCUS is Precision BioSciences' proprietary genome editing technology, distinguished by its ability to perform precise gene edits, such as gene elimination, insertion, and excision. The platform is based on a naturally occurring genome editing enzyme, I-CreI, from the algae Chlamydomonas reinhardtii.
Precision BioSciences, focusing on using ARCUS for genetic and infectious diseases, has a pipeline of in vivo gene editing candidates designed to deliver enduring cures for diseases with inadequate treatments. The company's work is supported by a comprehensive portfolio of more than 130 patents.
The information in this article is based on a press release statement from Precision BioSciences.
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