Press Release

Neurona Therapeutics Presents Encouraging Clinical Data from First-Ever Regenerative Human Cell Therapy Trial in Adults with Drug-Resistant Focal Epilepsy and Shows Supporting Manufacturing, Delivery, and Preclinical Data

– Early data from the first two patients treated with NRTX-1001 report a trend toward seizure suppression and that administration of the cell treatment has been well-tolerated thus far

– Pioneering cell therapy approach could provide a disease-modifying treatment for drug-resistant focal epilepsy

– Presentation at the ISSCR Boston International Symposium: Translating Pluripotent Stem Cell Discoveries to the Clinic: Preclinical, Manufacturing, and Regulatory Strategies for Success

San Francisco, CA, November 21, 2022 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, announced that Cory R. Nicholas, Ph.D., Neurona’s president and chief executive officer, was invited to present an overview of Neurona’s regenerative cell therapy technology, proprietary manufacturing process, cell delivery system, and preclinical data supporting its ongoing Phase I/II clinical trial of NRTX-1001 in people with focal epilepsy at the recent ISSCR Boston International Symposium: Translating Pluripotent Stem Cell Discoveries to the Clinic: Preclinical, Manufacturing, and Regulatory Strategies for Success. In his presentation on Saturday, November 19, 2022, Dr. Nicholas also provided an update on the first two patients treated in the groundbreaking clinical trial (NCT05135091), both of whom entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications. Following treatment with NRTX-1001, both patients have thus far seen encouraging signs of seizure reduction. 

“The early clinical results with NRTX-1001 in epilepsy are very encouraging, and we look forward to enrolling additional patients in the study,” said Dr. Nicholas. “NRTX-1001 is designed to be an off-the-shelf, one-time administration therapy with the potential to durably eliminate seizures and provide a new regenerative cell therapeutic approach in patients for whom anti-seizure medication has failed. It has the potential to be disease-modifying without the tissue-destructive procedural risks associated with lobectomy. Further, there are many who are not currently eligible for lobectomy surgery who may be eligible for NRTX-1001 in the future. We are sincerely grateful to everyone involved in the development of NRTX-1001, including the first participants in this pioneering study, their families, and the respective clinical site teams.”

Dr. Nicholas provided details on the manufacturing and characterization of NRTX-1001, which is an injectable suspension of high-purity inhibitory neurons, called interneurons, derived from human stem cells and designed to replace damaged neurons in the region of the brain where seizures originate. The cells are manufactured in Neurona’s in-house GMP facility, where its manufacturing personnel use proprietary methods to consistently guide the stem cells into a specific, pallial-type, lineage of interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). NRTX-1001 interneurons are cryopreserved and shipped to clinical sites to be used as an allogeneic, off-the-shelf investigative therapy.

Preclinical development of the product and the cell delivery system was also discussed. For clinical administration, NRTX-1001 is delivered using an established MRI-guided procedural approach performed in specialized epilepsy centers. Preclinical characterization of the NRTX-1001 product in epileptic models demonstrates that the administered cells migrate and functionally integrate into the targeted brain region, form connections with existing neurons, persist long-term, and eliminate seizures. 

In addition to the preclinical efficacy and NRTX-1001 characterization data supporting the clinical program, the presentation highlighted data from the first two adult patients with mesial temporal lobe epilepsy (MTLE) treated in Neurona’s clinical trial. The first patient had a 9-year history of seizures and in the six months prior to the administration of NRTX-1001, the patient experienced an average of 32 seizures per month, despite being on several antiepileptic medications. The patient received a single administration of NRTX-1001, the treatment was well tolerated, and there have been no serious or severe adverse events associated with the treatment to date. The patient reported four seizures during the first three months since receiving NRTX-1001. The second patient treated in the trial also had drug-resistant seizures, with an average of 14 seizures per month in the six months prior to treatment. This individual received NRTX-1001, returned home as planned the day following treatment and, in the first week post-treatment, had not experienced any serious or severe adverse events, or seizures.

The clinical trial is funded in part by the California Institute for Regenerative Medicine (DISC2-10525; TRAN1-11611; CLIN2-13355).

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)

Neurona’s multicenter, Phase I/II clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE, with five patients to be treated at a starting dose and five at a higher dose. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit (NCT05135091). The first part of the clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

About NRTX-1001

NRTX-1001 is a regenerative neural cell therapy candidate derived from human pluripotent stem cells. The fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

About Mesial Temporal Lobe Epilepsy 

An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite treatment with approved drugs, which means that there is a huge unmet medical need in this community. MTLE is the most common type of focal epilepsy in adults and primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. For people with seizures that are resistant to anti-seizure drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or ablated by laser, can be an option. However, the current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

About Neurona

Neurona’s regenerative cell therapy candidates have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit

Investor and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA