Neurona Reports Development of Investigational Novel Regenerative Cell Therapy Strategy for Drug-Resistant Focal Epilepsy

On October 5, 2023, Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, announced the publication of the development and characterization of a highly purified population of human stem cell-derived, pallial-specific MGE (medial ganglionic eminence) inhibitory interneurons for the potential treatment of focal epilepsy. In a preclinical study, administration of a single dose of the cells in a model of chronic drug-resistant MTLE (mesial temporal lobeepilepsy)resulted in durable and consistent suppression of focal seizures, as well as improvements in neuropathology and an increase in survival of the model. The study also demonstrated a potentially broad, safe, and effective dosing range in the preclinical model. The data support the development of Neurona Therapeutics’ regenerative cell therapy candidate, NRTX-1001, which is currently being evaluated in an ongoing Phase I/II clinical trial of a one-time dose in subjects with drug-resistant MTLE (NCT05135091). The preclinical data were published October 5, 2023 in Cell Stem Cell as a Clinical and Translational Report titled “Human Pallial MGE-Type GABAergic Interneuron Cell Therapy for Chronic Focal Epilepsy” by first authors Marina Bershteyn, Sonja Bröer, Mansi Parekh, Yves Maury, and colleagues and is available open-access online. The corresponding author is Cory R. Nicholas.

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