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Neurona - Chronic Disorders of the Nervous System
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 is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited neural cell therapies for single-dose targeted repair of the nervous system. Neurona’s discovery platform is powered by regenerative pluripotent stem cell technology and used to develop proprietary manufacturing processes that produce authentic neural cell therapy candidates. The company’s wholly-owned pipeline currently comprises cell types of a unique medial ganglionic eminence (MGE) lineage. Neurona’s lead product candidate, NRTX-1001, is composed of human MGE-type inhibitory neurons and is being investigated in a Phase I/II clinical trial for chronic focal epilepsy. Neurona is also advancing NRTX-1001 and its other cell therapy candidates for the treatment of additional chronic neurological disorders.
The MGE is a critical structure during brain development that generates a variety of specialized neuronal and glial cell lineages. The MGE is the predominant source of inhibitory neurons that secrete the neurotransmitter gamma-aminobutyric acid (GABA). The MGE also produces myelinating oligodendrocyte glial cells that insulate and support the forebrain. These MGE lineages are essential for the nervous system to function properly. Several neurological disorders are rooted in the imbalanced activity of neural circuits precipitated by improper development, dysfunction, or degeneration of MGE cell lineages, resulting in uncontrolled neural hyperexcitability and local excitotoxicity. Effective treatments for these neurological disorders have been lacking because traditional drugs often have imprecise systemic and diffuse neurological toxicities. In contrast, regenerative neural cell therapies that replace missing or damaged MGE neuronal and glial cell types can provide precise, targeted repair of affected neural circuits.
MGE cell therapy has broad therapeutic potential. Neurona has industrialized the production of human MGE cells using pluripotent stem cell technologies. From this regenerative human MGE cell platform, the company has developed proprietary methods for manufacturing authentic, fully-differentiated MGE-type neuronal and glial cell therapy candidates for targeted repair of the nervous system. Their unique ability to migrate, integrate, and modulate local neural circuits provides MGE cells with advantageous properties as a cellular therapeutic over most other neural cell lineages. Published proof-of-concept studies have demonstrated MGE cell therapy to be safe and effective in several preclinical models of neurological disorders.