Neurogene
Neurogene is a clinical-stage biotechnology company developing AAV9 gene therapies for rare neurological diseases, primarily NGN-401 for Rett syndrome using its proprietary EXACT transgene regulation technology. It is pre-revenue and publicly traded on NASDAQ as NGNE.
- Company typePublic
- Founded2018
- HeadquartersNew York, United States
- Headcount101–250
- GTM typeB2B
- OfferingServices
What Neurogene does
Neurogene is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases, with its lead program, NGN-401, targeting Rett syndrome and a secondary candidate, NGN-101, addressing CLN5 Batten disease. NGN-401 is an investigational AAV9 gene therapy designed to deliver a full-length human MECP2 gene under the control of the company's proprietary EXACT transgene regulation technology, administered as a one-time intracerebroventricular infusion. The EXACT platform is a trademarked technology published in Science Translational Medicine and is central to Neurogene's ability to regulate transgene expression, addressing the dose-titration challenges that have historically constrained MECP2-directed therapies. The company was founded in 2018 by Rachel McMinn, Ph.D., completed a reverse merger with Neoleukin Therapeutics in December 2023, and trades on NASDAQ as NGNE.
The company holds an unusually comprehensive set of regulatory designations on NGN-401 across the FDA (Breakthrough Therapy, RMAT, Fast Track, Orphan Drug, Rare Pediatric Disease), EMA (PRIME, ATMP), and UK MHRA (ILAP), and operates a 42,000 sq ft internal cGMP AAV manufacturing facility in Houston that supports clinical and potential early commercial supply. In June 2026 the company completed dosing of 25 participants in the Embolden registrational trial for NGN-401, and interim Phase 1/2 data have shown durable, multidomain functional improvements. The NGN-101 CLN5 Batten disease program has also completed Phase 1/2 enrollment with 13 participants.
Neurogene is pre-revenue and funds operations through equity capital. Following a $200M PIPE in November 2024, the company reported $243.2M in cash at the end of Q1 2026, which management states is sufficient to fund operations through Q1 2028. Net loss was $90.4M for FY2025 and $30.9M for Q1 2026, primarily reflecting R&D investment in the lead programs. Future revenue, if NGN-401 is approved, is expected to be generated through one-time sales to specialty treatment centers and hospitals, consistent with the AAV gene therapy commercial model. Leadership includes executives with prior experience at BioMarin, AveXis (Zolgensma), Alexion, and Avidity Biosciences, and the April 2026 appointment of a Chief Commercial Officer signals a transition from purely clinical execution toward pre-commercial buildout.
Neurogene firmographics
Firmographics- Name
- Neurogene
- Legal name
- Neurogene Inc.
- Website
- https://neurogene.com
- Company type
- Public
- Founded year
- 2018
- Operating status
- Operating
- Headcount range
- 101–250 employees
- Short description
- Neurogene is a clinical-stage biotechnology company developing AAV9 gene therapies for rare neurological diseases, primarily NGN-401 for Rett syndrome using its proprietary EXACT transgene regulation technology. It is pre-revenue and publicly traded on NASDAQ as NGNE.
- Ownership category
- akta.pro rank
Neurogene industry classification
Industry- Product category
- Gene Therapy for Rare Neurological Diseases
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- akta.pro primary industry
- Neurology/CNS Gene Therapies (HLAAACAH)
- akta.pro secondary industries
- Neurogenetics & Rare Neurologic Diseases (HLAKAIAO), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)
Keywords
Where Neurogene is headquartered
LocationHeadquarters
- HQ city
- New York
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Neurogene business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure
Revenue model
- Gene Therapy Product Sales: Neurogene is a clinical-stage biotechnology company with no current revenue. The company is developing NGN-401, an investigational AAV9 gene therapy for Rett syndrome, expected to be a one-time treatment. If approved, the company would generate revenue through product sales directly to healthcare providers and specialty centers.
Go-to-market motion2 records
Distribution channels3 records
Marketing channels6 records
Neurogene product offering
Product offeringCore offering
Neurogene develops investigational gene therapies for rare neurological diseases, with its lead candidate NGN-401 delivering the full-length human MECP2 gene via AAV9 under the proprietary EXACT transgene regulation technology for Rett syndrome, and NGN-101 for CLN5 Batten disease. Both are one-time intracerebroventricular (ICV) administered gene therapies targeting the root genetic cause of disease, supported by an in-house CGMP manufacturing capability in Houston, Texas.
Product overview
Neurogene is a clinical-stage biotechnology company developing genetic medicines for rare neurological diseases, with a primary focus on Rett syndrome. The company's core product pipeline consists of NGN-401 (an AAV9 gene therapy for Rett syndrome using the proprietary EXACT transgene regulation technology) and NGN-101 (a gene therapy candidate for CLN5 Batten disease). NGN-401 is administered as a one-time intracerebroventricular treatment and is currently being evaluated in the Embolden registrational clinical trial. The company maintains internal cGMP manufacturing capabilities in Houston, Texas.
Differentiator
Problem solved
Functional benefit
Products and services
- NGN-401 Investigational AAV9 gene therapy for females with Rett syndrome (ages 3 and older, post-regression) that delivers the full-length human MECP2 gene under Neurogene's EXACT transgene regulation technology, administered as a one-time intracerebroventricular (ICV) treatment to address the underlying genetic cause of Rett syndrome.
- NGN-101 Development candidate for CLN5 Batten disease containing conventional gene therapy elements designed to deliver the CLN5 gene packaged in an AAV9 capsid, currently in a Phase 1/2 clinical trial (CLN5-200) for patients with CLN5 Batten disease, a rare neurodegenerative lysosomal storage disease.
Quantifiable outcome
- Participants showed functional improvements including developmental milestones/skills gained across core clinical domains of Rett syndrome - hand function/fine motor, language/communication and ambulation/gross motor
- +3 more outcomes
Companies that use Neurogene
Customer profileNamed customers2 records
Segments2 records
Ideal customer profiles2 records
Neurogene technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Neurogene partnerships and signals
Strategic signalPartnerships
One partnership is on record.
- University of EdinburghcoreResearch collaboration with University of Edinburgh, a world leader in biomedical and translational research for neurodevelopmental diseases. The collaboration provides comprehensive research capabilities enabling the company and the university to accelerate scientific innovation to continue to improve upon conventional gene therapy. Chief Scientific Officer Stuart Cobb also leads a research team at the University of Edinburgh as Professor of Translational Neuroscience.
Scale indicators11 records
Recent moves7 records
Expansion highlights5 records
Neurogene competitors and assessment
Company assessmentDirect peers
- Taysha Gene Therapies: Clinical-stage gene therapy developer focused on AAV-based CNS treatments for rare monogenic neurological diseases, with overlapping technology approach (AAV9/AAV delivery to CNS) and rare disease regulatory strategy.
- uniQure: Clinical and commercial-stage AAV gene therapy company with approved CNS-directed therapy (Huntington's disease program) and pipeline spanning rare neurological disorders using similar AAV vector delivery platforms.
- Voyager Therapeutics: Gene therapy company developing AAV-based treatments for severe neurological diseases, using similar CNS-targeted delivery approaches and addressing monogenic CNS disorders with high unmet need.
Broad incumbents
- Sarepta Therapeutics: Commercial-stage genetic medicines company with multiple approved AAV gene therapies for rare neuromuscular diseases, providing a benchmark for CNS/neuromuscular gene therapy commercialization, pricing, and patient access dynamics.
- BioMarin Pharmaceutical: Established rare disease biotech with gene therapy programs (Roctavian for hemophilia A) and a baculovirus/Sf9 manufacturing platform that is highly analogous to Neurogene's dual-platform approach. Board member Robert Baffi led BioMarin's global manufacturing operations.
- Pfizer (Gene Therapy / Beqvez): Large pharmaceutical company with commercial-stage AAV gene therapy for hemophilia and prior CNS gene therapy programs through partnerships, providing a comparison point for commercial-scale gene therapy infrastructure and pricing.
- AveXis (Novartis): Novartis-owned gene therapy subsidiary that developed and commercialized Zolgensma for SMA via AAV9 delivery, providing the closest analog for CNS-directed AAV9 gene therapy commercialization that Neurogene aspires to replicate.
Emerging players
- Regenxbio: AAV gene therapy platform company whose NAV Technology Platform underpins several approved and clinical-stage CNS gene therapies, including partners developing treatments for rare neurological indications similar to Rett syndrome.
- Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for rare neuromuscular and CNS diseases, sharing platform technologies (AAV vectors) and orphan/rare disease regulatory pathways with Neurogene.
- Lexeo Therapeutics: Clinical-stage gene therapy company focused on CNS and cardiac rare diseases using AAV delivery, with pipeline overlap in monogenic neurological disorders and similar one-time treatment commercial model.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat4 records
Key risks5 records
Key highlights7 records
Customer concentration
Neurogene social profiles
Digital presenceNeurogene financial estimates
Financial estimateRevenue estimate
Valuation estimate
Neurogene leadership team
Management profileNumber of profiles
Profiles14 records
Neurogene funding detail
Funding detailFunding overview
Funding rounds4 records
Investors14 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Neurogene M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Neurogene
What does Neurogene do?
Neurogene develops investigational gene therapies for rare neurological diseases, with its lead candidate NGN-401 delivering the full-length human MECP2 gene via AAV9 under the proprietary EXACT transgene regulation technology for Rett syndrome, and NGN-101 for CLN5 Batten disease. Both are one-time intracerebroventricular (ICV) administered gene therapies targeting the root genetic cause of disease, supported by an in-house CGMP manufacturing capability in Houston, Texas.
Is Neurogene a public or private company?
Neurogene is a public company. It is classified as public and is currently operating.
When was Neurogene founded?
Neurogene was founded in 2018. It employs 101 to 250 people.
Where is Neurogene based?
Neurogene is headquartered in New York, United States, in the North America region.
How does Neurogene make money?
One revenue line is on record: gene Therapy Product Sales.
Who are Neurogene's main competitors?
Direct peers on record are Taysha Gene Therapies, uniQure and Voyager Therapeutics. Broad incumbents are Sarepta Therapeutics, BioMarin Pharmaceutical, Pfizer (Gene Therapy / Beqvez) and AveXis (Novartis). Emerging players are Regenxbio, Solid Biosciences and Lexeo Therapeutics.
Does Neurogene have an API?
No public API is recorded for Neurogene.
What industry is Neurogene in?
Neurogene's product category is Gene Therapy for Rare Neurological Diseases. Its primary akta.pro industry code is HLAAACAH, Neurology/CNS Gene Therapies, with a secondary code of HLAKAIAO, Neurogenetics & Rare Neurologic Diseases. Its NAICS code is 541714.