Neurona Therapeutics Presents Preclinical Data Supporting Regenerative Cell Therapy, … | Your money


The oral presentation will outline the design of the first-ever Phase 1/2 clinical trial of human cell regenerative therapy in epilepsy

The presentations will describe single-cell transcriptomic profiling of NRTX-1001 product composition and fate after administration in preclinical models

SAN FRANCISCO, June 17, 2022 (GLOBE NEWSWIRE) — Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological diseases, today announced the presentation of new drug discovery (IND) data targeting the company’s regulatory candidate inhibitory neuronal cell therapy, NRTX-1001 , support a first-in-human clinical trial for the treatment of drug-resistant focal epilepsy. The data will be presented in oral and poster presentations at the International Society for Stem Cell Research (ISSCR) Annual Meeting, being held June 15-18, 2022 in San Francisco, California.

“Supported by compelling preclinical data, Neurona has opened a clinical trial evaluating NRTX-1001 in adults with mesial temporal lobe epilepsy (MTLE),” said Cory R. Nicholas, Ph.D., Neurona’s President and Chief Executive Officer. “Currently, people living with drug-resistant MTLE have few options, including surgery to remove or ablate the affected temporal lobe. However, these tissue-destroying surgeries can have serious side effects and not all patients are suitable. NRTX-1001 has the potential to permanently restore neuronal inhibition to seizure foci after a single administration of cells.”

The data, presented by Marina Bershteyn, Ph.D., co-director of Neurona’s discovery research division, describes the characterization of NRTX-1001, a cellular therapeutic manufactured from human pluripotent stem cells using Neurona’s proprietary process at its proprietary manufacturing facility. NRTX-1001 cells were characterized prior to administration in functional and single-cell RNAseq assays, which confirmed high target purity of hippocampal/cortical interneurons, termed pallial-type GABAergic interneurons, consistent with the specific inhibitory cell lineage affected in the temporal lobe. After administration in preclinical models, RNAseq analysis of individual human NRTX-1001 nuclei showed adequate maturation of the cells into hippocampal/cortical interneuron sublineages.

Regardless, Dr. Nicholas will present data in an oral session showing that the NRTX-1001 interneurons stably persisted in a preclinical MTLE model, reproducibly eliminated focal seizures in the majority of the treatment group, reduced tissue damage in the temporal lobe, and improved survival rates. A dose-ranging study using the MTLE model identified a broad maximum effective dose range and no detectable dose-limiting toxicities. In addition, the MRI-guided clinical delivery system demonstrated consistent and safe deposition of the NRTX-1001 interneurons in a second preclinical model.

Based on these promising data, Neurona is initiating a first-ever clinical trial for the treatment of epilepsy using human cells. The study is a multi-center, open-label, dose escalation study followed by a randomized, controlled evaluation of the safety and efficacy of NRTX-1001 in people with drug-resistant MTLE.

Details of the presentations are as follows:

Title: RAPID DETECTION OF PALLIAL GABAERGIC INTERNEURON SUBTYPES BY SINGLE NUCLEI RNA SEQUENCING FOLLOWING PRECLINICAL TRANSPLANTATION OF NRTX-1001, A CELLULAR THERAPEUTIC UNDER CLINICAL DEVELOPMENT FOR EPILEPSY Authors: Marina Bershteyn, Robin Zhou, Luis Fuentealba, Geethakow Subramanyam, Daniel Meliz Sezan , Adrian Bates, Steven Havlicek, Yves Maury, Alessandro Bulfone, Gautam Banik, Catherine Priest, Cory R. Nicholas Poster #: 124 Date: Wednesday, June 15, 2022. 7:30-8:30 p.m. PT

Title: HUMAN INHIBITORY NEURON CELL THERAPY ENTERS PHASE I/II CLINICAL TRIAL IN CHRONIC FOCAL EPILEPSY Authors: Catherine Priest, Gautam Banik, David Blum, Alessandro Bulfone, Mansi Parekh, Philip Hampel, Hannah Kim, Andrew Adler, Luis Fuentealba, Michael Watson, Lee Seonok, Sonja Kriks, Steven Havlicek, Robin Zhou, Yves Maury, Marina Bershteyn, Cory R. Nicholas Session: Stem Cells – From Development to Therapy Date: Friday, June 17, 2022. 1:55 p.m. – 2:05 p.m pt

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE) Neurona’s Phase 1/2, multi-center clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. Patient recruitment is ongoing at epilepsy centers across the United States. For more information, please visit ( NCT05135091 ). The first phase of the clinical study is supported by a recently announced $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 derived from human pluripotent stem cells. The fully differentiated nerve cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Administered as a single dose, the human interneurons are designed to integrate and innervate in a targeted manner, providing long-term GABAergic inhibition to repair overexcitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE) MTLE primarily affects the inner structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common form of focal epilepsy in adults. For people who are resistant to antiseizure drugs, epilepsy surgery, in which the damaged temporal lobe is surgically removed or ablated with a laser, may be an option. However, current surgical options are not available or effective for everyone, are tissue destructive, and can have significant adverse effects.

About Neurona Neurona’s regenerative cell therapies have single-dose curative potential. Neurona is developing commercial allogeneic neuronal, glial and gene-edited cell therapy candidates to enable long-term repair of dysfunctional neuronal networks in multiple neurological diseases. For more information on Neurona, see

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