Participated · Equity · Feb 2024

Neurona Therapeutics is developing allogeneic, off-the-shelf regenerative neural cell therapy products intended to provide long-term targeted repair of the nervous system following a single administration. Its lead candidate, NRTX-1001, comprises GABAergic interneurons and is being evaluated in two ongoing open-label multicenter Phase 1/2 trials for drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (NCT05135091 and NCT06422923). The company presented updated Phase 1/2 data showing in a five-subject lower-dose cohort a median seizure reduction of 75% at six months, with four of five subjects reporting >50% reduction and two subjects reporting durable >95% reduction at 16 and 21 months; NRTX-1001 has been well tolerated and neuropsychological testing suggests absence of neurocognitive impairment. The FDA granted NRTX-1001 Regenerative Medicine Advanced Therapy (RMAT) designation in June 2024 based on these positive clinical data. Neurona plans to expand indications to neocortical focal epilepsy and other CNS disorders. Financially, the company has received multiple grants from the California Institute for Regenerative Medicine (CIRM), with cumulative CIRM commitment of $18.5M across five grants, and completed a $120M private financing in February 2024 co-led by Viking Global Investors and Cormorant Asset Management. Neurona Therapeutics is a San Francisco–based clinical-stage biotherapeutics company focused on developing regenerative, allogeneic, off-the-shelf cell therapy candidates with single-dose curative potential. Its candidates are designed to provide long-term repair of dysfunctional neural networks across multiple neurological indications. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing open-label, single-arm Phase I/II clinical trial for treatment of drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 also has potential application in Alzheimer’s disease and other disorders of the nervous system. Led by CEO Cory R. Nicholas, Ph.D., Neurona intends to use newly raised capital to advance its wholly-owned pipeline of off-the-shelf cell therapies. The company recently raised $120M and announced that Raymond Kelleher, M.D., Ph.D., of Cormorant will be joining its board of directors. Neurona Therapeutics develops neural cell therapies intended to provide long-term repair of dysfunctional neural networks in chronic neurological disorders. Its lead candidate, NRTX-1001, is a neuronal cell therapeutic derived from human pluripotent stem cells comprising GABA-secreting interneurons designed as a one-time administration to provide local long-term inhibition. The company is advancing wholly-owned, off-the-shelf neuronal, glial, and gene-edited cell therapy candidates across multiple indications, initially targeting temporal lobe (focal) epilepsy. Neurona plans a Phase 1/2a clinical study for NRTX-1001 planned to launch later this year. The company completed a $41.5M financing that will be used to advance the pipeline; total proceeds raised to date are $135 million. Neurona describes its platform as based on a novel neural cell lineage with single-dose curative potential. Neurona Therapeutics is a pre-clinical biotechnology company focused on the transplantation of selected neurons to treat intractable neurological diseases. The company develops regenerative cell-based therapeutics composed of unique cell compositions that can be precisely targeted and integrated into damaged neural circuits. Its approach emphasizes activity-dependent regulation once transplanted into host circuitry. Neurona was founded by neuroscientists and stem cell researchers from the University of California, San Francisco, including Arturo Alvarez-Buylla, Arnold Kriegstein, John Rubenstein and Cory Nicholas. The company is led by interim CEO Tim Kutzkey and launched with a $23.5M Series A financing. Neurona is based in South San Francisco, CA and remains in the pre-clinical stage.