Neucore Bio announced it has received a $350,000 STTR Phase I grant from the National Center for Advancing Translational Sciences (NCATS) at the U.S. National Institutes of Health to develop an exosome-based RNA therapy for Charcot-Marie-Tooth Disease Type 1A (CMT1A). The early-stage biotechnology company will evaluate its targeted exosome platform to deliver RNA-based therapeutics to treat this genetic neuromuscular disorder.
Addressing an Unmet Medical Need
CMT1A is a progressively debilitating genetic neuromuscular disorder caused by the duplication of the peripheral myelin protein 22 (PMP22) gene in Schwann cells. Currently, no approved therapies exist that can address this genetic defect to preserve function and quality of life for affected individuals. While RNA-based molecules have shown promise in silencing the PMP22 gene in early studies, limitations of viral delivery mechanisms have prompted researchers to evaluate non-viral alternatives.
"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 at Neucore Bio.
Proprietary Platform Technology
The NCATS-funded program will leverage Neucore's proprietary Fibroblast-Derived Engineering Extracellular Vesicles (FiXE™) platform, which has demonstrated in early studies the ability to systematically target Schwann cells. The research will focus on optimizing engineered extracellular vesicles (eEVs) to deliver microRNA constructs that regulate PMP22 overexpression. Planned Schwann cell co-culture experiments will demonstrate how effectively these programmed eEVs target Schwann cells and regulate PMP22 expression.
Engineered extracellular vesicle delivery approaches like exosomes have emerged as promising carriers of genetic cargo to target specific cell types because they are part of the body's natural mechanism for intercellular communication and cargo transfer between cells.
Advantages of Non-Viral Delivery
"EEVs represent a next-generation, non-viral delivery system capable of packing and delivering bulky genetic and molecular cargo for precise therapeutic applications. Their innate biocompatibility, ability to cross biological barriers, and low immunogenicity make them especially promising for repeat dosing in chronic conditions such as CMT1A," said Natalia Higuita-Castro, PhD, Associate Professor of Biomedical Engineering and Neurosurgery at The Ohio State University and Scientific Co-founder of Neucore.
Research Partnership and Clinical Path
The collaboration with The Ohio State University's Gene Therapy Institute brings extensive experience in translating gene therapies to the clinic. Daniel Gallego-Perez, PhD, Professor of Biomedical Engineering and Surgery at Ohio State and Scientific Co-founder of Neucore, noted that "this research effort aims to further optimize this platform for RNA-based therapeutics, and will provide critical proof-of-principle data needed to pursue validation studies that could ultimately bring this therapy into human trials."
The research is supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Number R41TR005758. Neucore Bio, based in Columbus, Ohio, has licensed novel exosome technology from The Ohio State University to power its discovery pipeline focused on genetic medicine and aesthetic dermatology solutions.
