Neurona Therapeutics is focused on developing innovative regenerative cell therapies aimed at repairing dysfunctional neural circuits associated with chronic neurological disorders. Their lead product candidate, NRTX-1001, utilizes human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells. This therapy is designed to target the seizure-onset zone in patients with drug-resistant focal epilepsy, enhancing GABAergic inhibition to reduce seizure activity and promote long-term neural network repair. The potential of NRTX-1001 extends beyond epilepsy, as ongoing preclinical studies are exploring its efficacy in treating Alzheimer’s disease by addressing subclinical electrical discharges in the temporal lobe, which may contribute to cognitive decline.

In addition to NRTX-1001, Neurona is developing a pipeline of allogeneic, off-the-shelf neural cell therapies, including myelinating glial cells and gene-edited cell types. The myelinating glial cell therapy aims to restore myelin in patients with dysmyelination disorders, leveraging a platform that simplifies the production of oligodendrocyte cells from pluripotent stem cells. Furthermore, the gene-edited cell therapies are engineered for targeted delivery of therapeutic factors to the central nervous system, overcoming challenges posed by the blood-brain barrier. This approach allows for precise engineering of cell therapies that can deliver complex therapeutic payloads sustainably, enhancing the potential for effective treatment of various chronic neurological conditions.

United States

Neurona is a biotherapeutics company developing neural cell therapies with single-dose curative potential for the treatment of chronic neurological disorders. Based on a novel neural cell lineage pioneered by the company’s scientific founders, Neurona has built a robust regenerative platform and is developing neuronal, glial, and gene-edited cell therapy candidates that provide long-term repair of dysfunctional neural networks for multiple neurological disorders.

Neurona Therapeutics

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