Armatus Bio acquired the CMT1A gene therapy that was developed by Dr. Kleopas Kleopa and Dr. Scott Harper in a previous CMTRF-funded project to advance it further as a therapeutic. To demonstrate this therapy could be a safe and effective treatment for CMT1A, Armatus Bio conducted a biodistribution study in a large animal model to test whether a drug administration method, called an intrathecal (IT) injection, allowed for sufficient delivery of the gene therapy to the Schwann cells of the peripheral nervous system while simultaneously overcoming dosage limitations, an important step for supporting clinical applicability of this gene therapy for CMT1A.

The results revealed that the gene therapy could be detected in the peripheral nerves following intrathecal administration and correlated with a reduction in PMP22 mRNA and protein, indicating that the gene therapy works and successfully reached cell types needed. The gene therapy was also well-tolerated with no significant safety concerns at the doses and timepoints investigated. Next steps include finalizing the clinical study design and completing the remaining IND-enabling toxicology and safety studies. This work will culminate in an IND submission to the FDA, marking a critical milestone in advancing ARM-101 as a potential therapeutic for CMT1A.

Dr. Scott Harper recently presented the findings from this project at the 2024 Global CMT Research Convention.