SAN FRANCISCO, July 11, 2025 (GLOBE NEWSWIRE) — Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today published a new peer-reviewed study in Neuron, a Cell Press journal, titled, “Human stem cell-derived GABAergic interneuron development reveals early emergence of subtype diversity and gradual electrochemical maturation”.
In the publication, the authors describe the long-term properties of a human stem-cell derived GABAergic interneuron cell therapy, the clinical-grade version of which, called NRTX-1001, is currently undergoing clinical evaluation by Neurona for drug-resistant epilepsy. Following the administration of human interneurons into healthy and epileptic mouse brains, the authors reported stable engraftment and consistent maturation into authentic pallial interneuron subtypes that functionally integrated with host circuitry.
Medial ganglionic eminence-derived pallial interneurons (MGE-pINs) are essential regulators of cortical circuits, and their dysfunction is associated with numerous neurological disorders. The human interneurons were developed from pluripotent stem cells as a potential treatment for drug resistant epilepsy. In this preclinical study, the authors analyzed xenografted human hMGE-pINs over the lifespan of mice in both healthy and epileptic environments using single nuclei RNA sequencing, histology, and electrophysiology.
The authors’ analysis revealed that 97% of grafted cells developed into on-target somatostatin (SST) and parvalbumin (PVALB) subtypes, including populations that exhibit selective vulnerability in epilepsy and Alzheimer’s disease. Moreover, the grafted hMGE-pINs demonstrated rapid emergence of subclass features, progressing through distinct transcriptional states sequentially involving neuronal migration, synapse organization, and membrane maturation.
“In this preclinical study, our team undertook systematic long-term molecular and functional characterization of human interneurons derived from pluripotent stem cells,” said Marina Bershteyn, Director of Discovery Research at Neurona Therapeutics and the study’s lead author. “By precisely specifying high-fidelity MGE interneurons synchronized at a post-mitotic migratory developmental stage before cell administration, we achieved unprecedented, durable control over the fate and function of the xenografted human interneurons. Our therapeutic strategy of administering fully-committed MGE pallial lineage-restricted interneurons ensures high on-target purity, consistent cell composition and functional engraftment.”
According to Cory Nicholas, CEO and co-founder of Neurona Therapeutics and the study’s co- senior author, “These preclinical data may help to explain the positive results demonstrated to date in Neurona’s ongoing Phase 1/2 clinical trial. We are delighted to share this publication with the scientific community, which provides valuable insight into human interneuron development, especially for certain interneuron subtypes that have thus far been elusive and difficult to study in the human brain.”
“Of particular note from the data generated by this preclinical study is that the hMGE-pINs developed physiologically into bona fide somatostatin and parvalbumin interneuron subtypes, including populations that are affected in Alzheimer’s disease,” said Jorge Palop, Associate Professor at the Gladstone Institute of Neurological Disease and University of California, San Francisco, and the publication’s co-senior author. “While NRTX-1001 is currently in development for the potential treatment of refractory focal epilepsy, these preclinical findings point to its potential in additional neurologic disorders, including Alzheimer’s, and warrant further investigation.”
In April, Neurona presented updated data from its unilateral MTLE trial at the AAN Annual Meeting, which included a 92% median reduction from baseline in disabling seizures during the 7-12-month efficacy endpoint after low-dose NRTX-1001 administration, improved quality-of-life scores, no adverse events related to the cell therapy, and no patient to date experiencing a persistent decline in cognition. Neurona plans to present extended durability data from the first ten unilateral MTLE patients treated with low (Cohort 1) and high (Cohort 2) doses of NRTX-1001, along with new results from the eight additional patients who were enrolled in the unilateral MTLE expansion cohorts, as well as early data from the first bilateral MTLE patients, at the American Epilepsy Society’s Annual Meeting in December 2025.
About Neurona Therapeutics
Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapies with the potential to provide long-term targeted repair of the nervous system following a single administration. Neurona’s lead product candidate, NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials: NCT05135091 for drug-resistant unilateral mesial temporal lobe epilepsy (MTLE), and NCT06422923 for drug-resistant bilateral MTLE, with expansion to neocortical focal epilepsy and other indications planned in the future. The Phase 1/2 MTLE clinical trials are supported by grants from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355 and CLIN2-17135). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024. Consistent with Neurona’s discussion with the FDA, the Phase 3 EPIC (EPIlepsy Cell Therapy) trial is planned to start in 2H 2025 and will provide primary support for submitting a potential future Biologics License Application (BLA). For more information about Neurona, visit: www.neuronatherapeutics.com.
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