Neucore Bio an early-stage biotechnology company, has been awarded a $350,000 STTR Phase I grant from the National Institutes of Health (NIH) to advance its research on an exosome-based genetic medicine for Charcot-Marie-Tooth Disease Type 1A (CMT1A). This progressively debilitating neuromuscular disorder is caused by the duplication of the PMP22 gene in Schwann cells, and there are currently no approved therapies that can address this genetic defect. The grant will fund a project that aims to demonstrate the targeted, non-viral delivery of an RNA-based therapy to these specific cells.

The company's approach utilizes its proprietary Fibroblast-Derived Engineering Extracellular Vesicles (FiXE™) platform to deliver microRNA constructs that regulate PMP22 overexpression. Exosomes are being explored as a promising non-viral alternative to traditional delivery mechanisms, as they are a natural part of the body's intercellular communication system, offering benefits such as biocompatibility, the ability to cross biological barriers, and low immunogenicity. Neucore Bio will partner with The Ohio State University, leveraging the institution's expertise in gene therapy. The funded study will focus on further optimizing the engineered exosomes and will conduct co-culture experiments to demonstrate their effectiveness in targeting Schwann cells and regulating PMP22 expression. This research is intended to provide critical proof-of-principle data needed to pursue further validation studies and, ultimately, human clinical trials.