Neurogene

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.

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

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

• Gene therapy development
• Rare neurological diseases
• AAV9 viral vectors
• Rett syndrome treatment
• CNS-targeted therapeutics

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. 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

Core 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

Named customers2 records

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

One partnership is on record.

• University of EdinburghcoreStrategic or Co-development PartnerResearch 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

Direct 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

• Xx.com/NeurogeneInc
• Facebookfacebook.com/NeurogeneInc
• LinkedInlinkedin.com/company/neurogene

Revenue estimate

Valuation estimate

Number of profiles

Profiles14 records

Funding overview

Funding rounds4 records

Investors14 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Neurogene