Neurona lands $120m to advance regenerative cell therapy pipeline

Company’s cell therapy candidates target long-term repair of dysfunctional neural networks associated with conditions including Alzheimer’s.

San Francisco-based biotech company Neurona Therapeutics has raised $120 million in a funding round to advance regenerative cell therapy candidates for the treatment of neurological disorders. The company’s investigational allogeneic, off-the-shelf cell therapy candidates aim to provide long-term repair of dysfunctional neural networks associated with conditions including epilepsy and Alzheimer’s disease.

The company’s lead candidate, NRTX-1001 is a regenerative neural cell therapy, derived from human pluripotent stem cells, consisting of fully differentiated interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).

The round was co-led by Viking Global Investors and Cormorant Asset Management, with participation from The Column Group, as well as new and existing investors, and comes on the heels of promising early clinical data from the company’s lead program in epilepsy.

Currently undergoing an open-label, single-arm Phase I/II clinical trial, NRTX-1001 is being assessed for its effectiveness in treating drug-resistant mesial temporal lobe epilepsy. The therapy also has potential applications in neurodegenerative disease, with the company claiming it presents a novel therapeutic strategy for inhibitory neuron therapy in Alzheimer’s.

“As a neurologist, I am particularly encouraged by the data generated thus far, suggesting that NRTX-1001 has the potential to provide seizure control and preserve neurocognitive function, which would be a game-changer for the field,” said Dr Raymond Kelleher, managing director of Cormorant and a neurologist at Massachusetts General Hospital, who joins Neurona’s Board.

In addition to its neural cell therapies, Neurona is also advancing a myelinating glial cell therapy platform, which expedites the production of human oligodendrocyte cells from pluripotent stem cells. The MGE oligodendrocyte candidate is under evaluation for its disease-modifying activity in preclinical studies exploring multiple undisclosed potential future indications.

Beyond its unmodified cell therapy candidates, Neurona is also exploring the development of genetically modified, or gene-edited, cell types for targeted delivery of therapeutic factors to the central nervous system. Overcoming the challenge of the blood-brain barrier, Neurona’s cell therapies are designed to integrate and persist long-term within the CNS, providing a potential solution for delivering therapeutic agents, especially for large molecules.

“This funding will support ongoing and planned clinical studies of NRTX-1001 for drug-resistant epilepsies and Alzheimer’s disease, as well as the advancement of our other cell therapy candidates towards the clinic for additional neurological indications,” said Dr Cory R. Nicholas, CEO of Neurona.

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