NGN-401 (Rett syndrome)
“10-K Item 1: 'Our most advanced program, NGN‑401, is in Phase 3 clinical development for the treatment of Rett syndrome.'”
Updated
The most significant concentration Neurogene discloses is NGN-401 (Rett syndrome), classified HIGH by disclosed size. Below: the full set from the latest 10-K — verbatim quotes, filing references, and a synthesis of what these exposures mean together.
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Source: Neurogene’s SEC Form 10-K filed — view the filing on SEC EDGAR ↗
Each card carries a disclosed-size chip (HIGH / MEDIUM / LOW — how large the exposure is as a share of revenue, not how dangerous it is) and a nature tag: Built-in(the company’s own model, geography, or products) or Outside party (an external customer, supplier, or distributor it relies on).
“10-K Item 1: 'Our most advanced program, NGN‑401, is in Phase 3 clinical development for the treatment of Rett syndrome.'”
Neurogene's disclosed concentration is narrow and single-pointed: the company identifies NGN-401, its program for Rett syndrome, as its most advanced candidate, now in Phase 3 clinical development. This is a structural concentration rather than an idiosyncratic customer or supplier dependency — it reflects the standard profile of a clinical-stage biotech that has advanced only one asset into late-stage trials, not a counterparty-specific vulnerability. With no other concentration items disclosed in the sourced filing text, there is no visible diversification across programs, geographies, manufacturing partners, or payers to offset this exposure. The practical implication is that the investment case tied to this risk category rests almost entirely on NGN-401's continued clinical progress and eventual regulatory outcome; any setback in that single Phase 3 program would fall directly on the company's near-term prospects, since the filing does not point to other assets that could absorb the impact. Because only one structural item is disclosed here, there is little basis in the source material to weigh competing or offsetting exposures — this is best read as a standard single-asset biotech concentration profile rather than a multi-pronged risk picture.
For the engine’s reasoning on NGNE’s current verdict — including which dimensions drove the score — see the per-dimension breakdown.
| Symbol | Name | HIGH | MEDIUM | LOW | Total |
|---|---|---|---|---|---|
| ACAD | ACADIA Pharmaceuticals Inc. | 2 | 0 | 0 | 2 |
| ABUS | Arbutus Biopharma Corporation | 1 | 1 | 0 | 2 |
| ABSI | Absci Corporation | 1 | 0 | 0 | 1 |
| NGNE● | Neurogene Inc. | 1 | 0 | 0 | 1 |
| ABCL | AbCellera Biologics Inc. | 0 | 0 | 0 | 0 |
| ACHV | Achieve Life Sciences, Inc. | 0 | 0 | 0 | 0 |
Concentration counts reflect items disclosed in each peer’s most recent 10-K; disclosed-size classification uses TrendMatrix’s internal 10-K extraction taxonomy.