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Neurogene Inc. is a biotechnology company focused on developing gene therapies for rare, complex neurological diseases. Utilizing proprietary EXACT transgene regulation technology and in-house manufacturing, it advances clinical-stage programs like NGN-401 for Rett syndrome and pipeline candidates for other neurodegenerative disorders. The company leverages collaborations with academic institutions, targeting unmet medical needs with innovative, scalable genetic medicines delivered via optimized routes to the central nervous system.
This description was generated via AI from an annual report. Updated about 1 year ago.