Press Release

Neurona Therapeutics Awarded $8 Million by California Institute for Regenerative Medicine (CIRM) to Support Phase 1/2 Clinical Trial of NRTX-1001 for Chronic Focal Epilepsy

– CIRM grant awarded to accelerate clinical trial of NRTX-1001, a promising regenerative neuronal cell therapy in adults with drug-resistant seizures

– NRTX-1001 is the first human cell therapy candidate to enter clinical investigation for the treatment of epilepsy

San Francisco, CA, May 26, 2022 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced that it has been awarded a $7,999,999 grant by the California Institute for Regenerative Medicine (CIRM) to support a dose-escalation Phase 1/2 clinical trial of NRTX-1001, an inhibitory cell therapy that is being evaluated for safety and efficacy in people with drug-resistant mesial temporal lobe epilepsy (MTLE). 

“Our regenerative cell therapy approach has the potential to repair the hyperexcitable neural networks that underlie focal epilepsy,” said David Blum, M.D., Neurona’s chief medical officer. “About a third of individuals with epilepsy have drug-resistant seizures. Removal or ablation of the affected temporal lobe can be an option, but many people with epilepsy are not eligible or interested in a tissue-destructive procedure. If successful, NRTX-1001 cell therapy will provide patients a non-destructive option for the treatment of their seizures.”

Cory R. Nicholas, Ph.D., Neurona’s president and chief executive officer, added, “We appreciate the continued support and validation of our work by CIRM as we pursue our bold vision of creating novel regenerative cell therapies that have the potential to profoundly improve the quality of life for people with chronic neurologic disorders.”

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

Neurona’s multi-center, Phase 1/2 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. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit (NCT05135091).

About NRTX-1001

NRTX-1001 is a regenerative neural cell therapy 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 hyperexcitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE)

MTLE primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy in adults. For people 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 therapies have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that provide long-term integration and repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit


Neurona Therapeutics

Investor and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA