Clinical trials
catalyst calendar across sponsors →Studies where NGNE is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
3 interventional · 3 observational
Held by 8 tracked-famous managers (Balyasny Asset Management L.P., CITADEL ADVISORS LLC, Point72 Asset Management, L.P., MILLENNIUM MANAGEMENT LLC, JANE STREET GROUP, LLC, +3 more) · FINRA short interest 16.1 days to cover (settled 2026-07-31) · government filings 180d: none disclosed
| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett SyndromeNCT05898620Efficacy of NGN-401Non-randomized · Single-masked · Treatment | Phase 3 | Active, not recruiting | Genetic: NGN-401 | Rett Syndrome | 33 | Dec 2029 | ≤ 1,230 d | 2026-06-10 |
| Gene Therapy Study for Children With CLN5 Batten DiseaseNCT05228145Incidence of Treatment Emergent Adverse Events (TEAEs)Non-randomized · Open-label · Treatment | Phase 1/2 | Active, not recruiting | Genetic: NGN-101 | Neuronal Ceroid Lipofuscinosis CLN5 | 6 | Nov 2028 | ≤ 834 d | 2024-08-12 |
| A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)NCT03822650observationalUnified Batten Disease Rating Scale (UBDRS) | — | Completed | — | Ceroid Lipofuscinosis, Neuronal 5 | 37 | 2024-05-30actual | — | 2024-06-27 |
| NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory CancerNCT04659629Recommended phase 2 dose (RP2D) for NL-201 (Parts 1 and 2)Non-randomized · Open-label · TreatmentStudy Protocol and Statistical Analysis Plan ↗ | Phase 1 | Completed | Drug: NL-201, Pembrolizumab Injection [Keytruda] | Solid Tumor, Advanced Solid Tumor | 59 | 2022-12-15actual | — | 2024-10-01 |
| A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)NCT03810508observationalCharcot Marie-Tooth Pediatric Scale (CMTPedS) | — | Terminatedsponsor's stated reason: Discontinued CMT4J development program | — | Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV | 21 | 2022-02-15actual | — | 2022-03-31 |
| A Natural History Study of AspartylglucosaminuriaNCT03853876observationalNeuropsychological Testing | — | Terminatedsponsor's stated reason: Discontinued AGU development program | — | Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency, Lysosomal Storage Diseases | 8 | 2020-10-15actual | — | 2022-04-12 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19