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Neurona Therapeutics

South San Francisco, California, United Statesneuronatherapeutics.com ↗

Ownership: Private
Development stage: Phase 1

Overview

Neurona’s lead candidate NRTX‑1001 has shown promising results in ongoing open-label Phase I/II trials for drug-resistant MTLE. In the first cohort of five subjects, a median reduction of disabling seizures by up to ~92% was observed at the primary efficacy endpoint period. Two patients have maintained >95% seizure reduction more than one year post-treatment; one patient has been seizure-free for six months after discontinuing immunosuppression. No major safety concerns or cognitive decline reported so far. A pivotal Phase III EPIC trial will begin enrollment in late 2025 as a basis for BLA submission.[5][7][9]

Therapeutic areas: Neurology
Modalities: Allogeneic cell therapyNeural cell therapy (GABAergic interneurons)Gene-edited cell therapies (in pipeline)
Industry: Biotech

Drug pipeline

1 asset

NRTX-1001

Partnerships

California Institute for Regenerative Medicine (CIRM) – grant funding/support[5][9]Academic collaborations with University of California San Francisco founders/researchers[6]

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