Neurogene is a clinical-stage biotechnology company focused on developing life-transforming gene therapies for rare neurological diseases that affect the central nervous system. The company's pipeline targets severe pediatric genetic disorders including Rett syndrome, a devastating neurological condition primarily affecting girls, and other rare diseases where current treatment options are limited or nonexistent. Neurogene's approach utilizes adeno-associated virus (AAV) gene therapy vectors designed to cross the blood-brain barrier and deliver functional copies of disease-causing genes to cells throughout the nervous system. Their clinical development programs advance novel gene therapies through rigorous testing to evaluate safety, optimal dosing, and therapeutic benefit in patients with these rare conditions. Neurogene employs advanced manufacturing capabilities, biomarker development, and clinical trial designs specifically adapted for rare neurological diseases where traditional endpoints may not apply. The company collaborates with leading academic medical centers, patient advocacy organizations, and regulatory agencies to advance therapies toward approval while supporting patients and families affected by these conditions. Backed by prominent life sciences investors and strategic partners, Neurogene represents cutting-edge innovation in gene therapy targeting significant unmet needs in rare neurological diseases where new treatments could dramatically improve patient outcomes and quality of life.
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