COMPANY

Neurogene

HQ: New York, NY source · neurogene.com ↗ · Mid-Atlantic

Neurogene is focused on developing genetic medicines for patients affected by rare neurological diseases. From company website Source · checked 2026-10-19

Sources for this description (1)

Neurogene is focused on developing genetic medicines for patients affected by rare neurological diseases.
- “Neurogene is focused on developing life-changing genetic medicines for patients and their families affected by rare, devastating neurological diseases.” https://www.neurogene.com/pipeline/ (2026-10-19)

Modality: gene therapy

Keywords: neurology gene therapy Rett Syndrome Neuronal Ceroid Lipofuscinosis CLN5 Neuronal 5 Ceroid Lipofuscinosis Aspartylglucosaminuria Aspartylglucosamidase (AGA) Deficiency Lysosomal Storage Diseases Charcot-Marie-Tooth Disease Hereditary Motor and Sensory Neuropathy IV NGN-401 NL-201 NGN-101

Location: New York, NY · source · Oct 15, 2026

Profile checked Oct 10, 2026

$200.0MRaised (2 yrs)6Trials (company is sponsor)

Timeline

Nov 4, 2024 Raise Private placement: $200M (total, as published), announced Nov 4, 2024. Source: SEC filing. source ↗
Jun 13, 2023 Trial start Phase 3: A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome source ↗
Jan 31, 2022 Trial start Phase 1/Phase 2: Gene Therapy Study for Children With CLN5 Batten Disease source ↗
Apr 26, 2021 Trial start Phase 1: NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory Cancer source ↗
Jul 18, 2019 Trial start —: A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J) source ↗
Apr 18, 2019 Trial start —: A Natural History Study of Aspartylglucosaminuria source ↗
Mar 13, 2019 Trial start —: A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5) source ↗

Upcoming milestones

2028-11 (ESTIMATED) · Phase 1/Phase 2 · Gene Therapy Study for Children With CLN5 Batten Disease · NCT05228145
2029-12 (ESTIMATED) · Phase 3 · A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome · NCT05898620

Estimated primary completion per ClinicalTrials.gov. Dates move.

Funding

Raised: $200.0M. Last 2 years.

Nov 4, 2024 Private placement: $200M (total, as published), announced Nov 4, 2024. Source: SEC filing. source ↗

Clinical trials (lead sponsor) 6

Record	Title	Phase	Status	Start	Primary completion
NCT05898620	A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome	Phase 3	Active, not recruiting	Jun 13, 2023	2029-12 (estimated)
NCT04659629	NL-201 Monotherapy and in Combination With Pembrolizumab in Patients With Relapsed or Refractory Cancer	Phase 1	Completed	Apr 26, 2021	Dec 15, 2022 (actual)
NCT05228145	Gene Therapy Study for Children With CLN5 Batten Disease	Phase 1/Phase 2	Active, not recruiting	Jan 31, 2022	2028-11 (estimated)
NCT03822650	A Natural History Study of Neuronal Ceroid Lipofuscinosis Type 5 (CLN5)	Not listed	Completed	Mar 13, 2019	May 30, 2024 (actual)
NCT03853876	A Natural History Study of Aspartylglucosaminuria	Not listed	Terminated	Apr 18, 2019	Oct 15, 2020 (actual)
NCT03810508	A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)	Not listed	Terminated	Jul 18, 2019	Feb 15, 2022 (actual)