{"schema":"rootz.ai/sec-company/v1","layer":1,"cik":"0001404644","name":"Neurogene Inc.","summary":"Neurogene Inc. is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases. Its proprietary EXACT (Expression Attenuation via Construct Tuning) technology uses microRNA-based genetic circuits to regulate transgene expression. Its lead program, NGN-401, is in a registrational trial (Embolden) for Rett syndrome, delivered via one-time intracerebroventricular AAV injection. The company operates its own cGMP manufacturing facility in Houston, Texas.","people":[],"products":[{"name":"NGN-401","type":"platform","description":"AAV-based gene therapy using EXACT technology for Rett syndrome, delivered via intracerebroventricular injection; in Phase 3 registrational trial (Embolden)."},{"name":"EXACT Technology","type":"platform","description":"Proprietary microRNA-based transgene regulation platform designed to control gene expression and widen therapeutic windows for neurological diseases."},{"name":"NGN-101","type":"platform","description":"Gene therapy program for CLN5 Batten disease; discontinued in November 2024 due to rarity and lack of streamlined FDA registrational pathway."}],"partnerships":[{"partner_name":"University of Edinburgh","partnership_type":"development","description":"Master Research Collaboration (MCA) supporting research and pipeline development; provides option to in-license product candidates from Dr. Stuart Cobb's laboratory. 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