Funded project aims to demonstrate targeted non-viral delivery of RNA to Schwann cells, which play a critical role in the pathogenesis of neuromuscular disease

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.”

As part of the study, Neucore will leverage its proprietary Fibroblast-Derived Engineering Extracellular Vesicles (FiXE™) platform, which has demonstrated in early studies the ability to systematically target SCs. The NCATS-funded program will further optimize the eEVs to deliver microRNA constructs that regulate PMP22 overexpression. Planned SC co-culture experiments will demonstrate how effectively these programmed eEVs target SCs and regulate PMP22 expression.

“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.

“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,” added Daniel Gallego-Perez, PhD, Professor of Biomedical Engineering and Surgery at Ohio State and Scientific Co-founder of Neucore.

About Neucore Bio

Neucore Bio, Inc., is an early-stage biotechnology company advancing nonviral, engineered exosomes to power the next generation of genetic medicine and aesthetic dermatology solutions. The company’s proprietary platform harnesses the innate programmability of fibroblasts and the natural intercellular signaling of exosomes to deliver nucleic acid therapeutics for disease modification and skin rejuvenation - unlocking new era of performance-driven genetic medicine and functional skincare. Based in Columbus, Ohio, Neucore has licensed novel exosome technology from The Ohio State University to power its disruptive discovery pipeline. For more information, visit www.neucorebio.com.

This research is supported by the National Center for Advancing Translational Sciences of the National Institutes of Health under Award Number R41TR005758. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

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