Neucore Bio, an early-stage biotechnology company advancing the therapeutic potential of non-viral, engineered exosomes, today announced the receipt of a $350,000 STTR Phase I grant from the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health (NIH) to evaluate the Company’s targeted exosome platform to deliver an RNA-based therapy to treat Charcot-Marie-Tooth Disease Type 1a (CMT1A).
CMT1A is a progressively debilitating genetic neuromuscular disorder caused by the duplication of the peripheral myelin protein 22 (PMP22) gene in Schwann cells (SCs). Currently there are no approved therapies that can address this genetic defect to preserve function and quality of life for affected individuals. RNA-based molecules have shown promise in silencing the PMP22 gene in early studies, yet limitations of viral delivery mechanisms warrant evaluation of non-viral alternatives. Engineered extracellular vesicle (eEV)-delivery approaches like exosomes have emerged as promising carriers of genetic cargo to target specific cell types, like SCs, because they are part of the body’s natural mechanism for intercellular communication and cargo transfer between cells.
“We are humbled to receive this NIH award as it recognizes the true potential of our novel eEV delivery platform to overcome the challenge of targeting specific cells like SCs; this is foundational to unlocking the next generation of genetic medicines and solving important unmet needs in patient care,” said Silvia Duarte-Sanmiguel, PhD, Research and Development Director, Neucore Bio. “Importantly, the partnership with The Ohio State University is critical given its Gene Therapy Institute, which brings a wealth of experience in translating gene therapies to the clinic. Leveraging our combined strengths, we are working to advance our platform toward clinical application.”