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Neurogene

NGNE Rare disease Lead: Phase 3 4 Biotech Distilled articles ↓ 20 filings & releases ↓ Cash $112M (Jun 30, 2026) ↓ 4 verdicts ↓ 2 timing quotes ↓
Neurology Gene Therapy

Market cap $506M 20.0M shares (10-Q, Aug 7, 2026) × $25.31 close Nov 6, 2026

Most recent annual filing: 2026-03-24

Catalyst timeline

1 trackedQuarter catalyst calendar →
  1. 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)

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)

Capital (as filed)

Cash
$112M cash and cash equivalents · Jun 30, 2026 (10-Q)
Short-term investments
$113M Jun 30, 2026
Operating cash flow
−$23.8M quarter Mar 31, 2026 → Jun 30, 2026, year-to-date difference (10-Q)
As stated in coverage
$135M cash, runway Q1 2029 per the update of Jul 2, 2026 — the company's own words as read there, not the filed figure

Offering documents (1)

Cash, investments and operating cash flow are the company's own XBRL facts as filed with the SEC, each shown with its period end and tag. As-stated figures come from the company's own words as read in Biotech Distilled coverage, dated, and routinely count securities and planned financing that a balance sheet does not. Offering rows are the SEC cover pages as filed. Not investment advice. Figures as of Nov 7, 2026.

Biotech Distilled coverage (4)

Articles open on Substack. Some are subscriber-only; those marked ★ Distilled Pro are for Distilled Pro members.

Verdict history (4 calls)

Every verdict Biotech Distilled has published on Neurogene, oldest first — the full history, never a selection. How calls are scored: the track record.

  1. 2026-06-29 Watch Neurogene (NGNE) — Scientific Deep Dive for NGN-401 and the EXACT Gene Therapy Platform Differentiated EXACT-platform science and elite syndicate, but open-label single-arm binary against a cleaner competitor with a pediatric death in the file keeps it off a Buy until Embolden de-risks.
  2. 2026-06-30 Speculative buy speculative UPDATE: Neurogene (NGNE) — The Phase 1/2 Print Lands, and the Tape Yawns Phase 1/2 back-half cohort converted to 8/10 responders at 12+ months with 30-month durability and clean safety, pre-de-risking Embolden; asymmetry now more obviously skewed upside for those who can hold the binary.
  3. 2026-06-30 Speculative buy speculative UPDATE: Neurogene (NGNE) — The Raise-Into-Strength I Told You To Watch For Opportunistic raise extends runway to Q3 2028 past the binary with launch-prep funding; specialist syndicate appears positioned to participate; thesis unchanged, only plumbing shifted.
  4. 2026-07-02 Speculative buy speculative UPDATE: Neurogene (NGNE) — The Raise Prints: $30, $134.8M All-In, Runway to Q1 2029 Raise priced at $30 with full same-day greenshoe exercise signals demand; runway extended to Q1 2029 providing genuine launch-prep buffer past the 2H 2027 Embolden binary; financing overhang removed.

Every verdict Biotech Distilled has published on this company, in order, with the post it came from — the full history, never a selection. Position figures are the same ones shown on /track-record, computed by the same method. Editorial opinion, not investment advice.

Timing, in the company's words (2 quotes from 2 filings)

Sentences quoted verbatim from the company's SEC filings, newest filing first. Each sits under the filing it appeared in; the "matched to" tag names the calendar row it was read for.

Timing sentences quoted from the company's own SEC filings, each under the filing it appeared in. The 'matched to' tag is the calendar row the sentence was read for; the filing is the source. Not investment advice.

Filings & releases (20)

SEC filings from EDGAR and press releases captured from the company's investor-relations site, newest first. Releases captured from Jul 28, 2026; filings from March 2026. Summaries come from the BD update that read the document; "BD extracted" counts point at the pipeline table above. All companies →

7 more filings (13G holder notices, S-8, proxy material, SEC correspondence …)