NGN-401
Gene Therapy Target: MECP2
Neurogene
Most recent annual filing: 2026-03-24
Pipeline (1 active programs)
Technology platforms
EXACT · University of Edinburgh
Expression Attenuation via Construct Tuning — proprietary microRNA-based transgene regulation technology designed to deliver controlled therapeutic transgene expression levels, widen the therapeutic window for dosage-sensitive neurological diseases, and avoid toxicity from variable gene expression. Delivery-agnostic; compatible with viral and non-viral platforms. Developed in collaboration with the University of Edinburgh. Disease focus: complex rare neurological diseases not addressable by conventional gene therapy.
EXACT
Proprietary transgene regulation technology designed to deliver consistent, tightly controlled protein expression on a cell-by-cell basis; used in AAV gene therapy programs for neurological diseases including Rett syndrome
Targets: MECP2
Discontinued (1)
- NGN-101 — CLN5 Batten Disease · RMAT application denied; lack of streamlined registrational pathway with FDA given rarity of disease; company does not expect to advance the program
Biotech Distilled coverage (4)
- 2026-07-02 UPDATE: Neurogene (NGNE) — The Raise Prints: $30, $134.8M All-In, Runway to Q1 2029
- 2026-06-30 UPDATE: Neurogene (NGNE) — The Phase 1/2 Print Lands, and the Tape Yawns
- 2026-06-30 UPDATE: Neurogene (NGNE) — The Raise-Into-Strength I Told You To Watch For
- 2026-06-29 Neurogene (NGNE) — Scientific Deep Dive for NGN-401 and the EXACT Gene Therapy Platform
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