Neurogene

NGNE Rare disease Lead: Phase 3 4 Biotech Distilled articles ↓

Most recent annual filing: 2026-03-24

Catalyst timeline

NGN-401 Ph1 interimMid-2026NGN-401 Ph1 interim, Mid-2026, material
NGN-401 corporate updateMid-2026NGN-401 corporate update, Mid-2026, routine
Embolden corporate updatemid-2026Embolden corporate update, mid-2026, routine
NGN-401 topline2H 2027NGN-401 topline, 2H 2027, thesis-defining

Bar width shows how precise the guidance is — a pin is a stated date, a wide band a six-month window. Deeper blue means more of the thesis rides on the event; it is not a view on the stock.

Pipeline (1 active programs)

NGN-401

Gene Therapy Target: MECP2

Rett Syndrome Phase 3 Phase 1/2 trial intracerebroventricular NCT05898620

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 🔒 Paid
2026-06-30 UPDATE: Neurogene (NGNE) — The Phase 1/2 Print Lands, and the Tape Yawns 🔒 Paid
2026-06-30 UPDATE: Neurogene (NGNE) — The Raise-Into-Strength I Told You To Watch For 🔒 Paid
2026-06-29 Neurogene (NGNE) — Scientific Deep Dive for NGN-401 and the EXACT Gene Therapy Platform 🔒 Paid

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