Neurona Therapeutics products

Chronic Disorders of the Nervous System are Prime Indications for Regenerative Medicine. Neurona is a clinical-stage biotherapeutics company developing regenerative cell therapies that permanently integrate into neural circuits, replace affected cells, and rebalance dysregulated electrical activity to repair the nervous system. Neural cell therapies have the potential to achieve long-lasting disease amelioration for chronic neurological disorders that lack effective treatment options, from a single, one-time dose. In this way, neural cell therapies can be curative.

Neurona’s lead product candidate, NRTX-1001, comprises human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells. NRTX-1001’s first indication is for the treatment of drug-resistant focal epilepsy, which is a major unmet need affecting approximately one-third of people living with epilepsy. Epileptic seizures are characterized by imbalanced neural activity and insufficient GABAergic inhibition, resulting in hyperactive neural networks. Refractory seizures can be disabling and significantly impair quality of life, and there are few effective options currently available to those suffering from chronic epilepsy. For some with refractory focal epilepsy, a surgical lobectomy to remove or ablate the seizure-onset zone can be an option but risks significant adverse effects due to the tissue-destructive nature of the surgery.

A wholly-owned pipeline of novel cell therapy products. Neurona is developing allogeneic, off-the-shelf neural cell therapies for multiple chronic neurological disorders. The pipeline includes neuronal, glial, and gene-edited cell therapy candidates led by NRTX-1001, a first-in-class inhibitory neuron therapy for epilepsy.

The central nervous system (CNS) contains both excitatory cells (primarily neurons that express glutamate) and inhibitory cells (primarily neurons that express GABA). Normal brain and spinal cord function depends on a delicate balance between these two types of neurons. This balance is normally established during embryonic development and just after birth when the nervous system is still assembling and developing neurologic connections.