Neurogene : Corporate Presentation March 2025

Published: 2025-03-24 16:41:13 ET NGNE

Corporate Presentation

March 2025

Neurogene is a Differentiated Clinical-Stage Company Utilizing Technology to Treat Complex Neurological Diseases

Novel EXACT technology designed to overcome key limitations of conventional gene therapy

$

Pipeline addresses attractive market opportunities, including Rett syndrome

Internal manufacturing provides financial and strategic pipeline flexibility

2H:27 cash runway enables operations beyond clinical inflection points

EXACT: Expression Attenuation via Construct Tuning

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Neurogene Clinical Stage Pipeline

Transgene Regulation CNS + Ocular Delivery

Product

Indication

IND* Enabling

Phase I/2

Pivotal

Near-Term

Candidate

Expected Milestones

Registrational Trial Plans Update

NGN-401

Rett Syndrome

1H:25

Additional Interim Data

2H:25

NGN-101

CLN5 Batten Disease

Evaluating Opportunities for

Program

*IND = investigational new drug

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Wholly Owned and Fully Integrated In-House AAV Manufacturing

42,000 sq ft facility in Houston, with 6,000 sq ft of cleanrooms

Analytical

Development

Quality

Control

GMP

Manufacturing

Toxicology Batch

Manufacturing

Process

Development

Quality

Assurance

Current research and clinical-grade manufacturing capabilities are designed for commercial-grade product to avoid potential future comparability challenges

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NGN-401 for Rett Syndrome

Leveraging EXACT transgene regulation technology

Rett Syndrome - Devastating Disorder with High Unmet Need

Genetics

Compelling Market Opportunity

• U.S. prevalence - ~6,000-9,000 patients

High Unmet Need

• There are no approved treatments that address root cause of disease

U.S. prevalence estimate based on published incidence rates; Laurvick CL, et al. J Pediatr 2006;148(3):347-35.

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WW incidence estimate based on published incidence rates; Pini G, et al. Orphanet Journal of Rare Diseases (2016) 11:132.

Rett Syndrome Treatment Requires Tight Transgene Regulation

Rett Syndrome*

MeCP2

Duplication Disorder**

Too little

gene expression drives disease

Balanced

treatment goal

Too much

gene expression drives disease

~50% of cells express WT levels of MeCP2

~100% cells express 2x MeCP2 levels

~50% are MeCP2 deficient

NGN-401 is designed to deliver therapeutic levels of MeCP2 to deficient cells while

maintaining a non-toxic level in unaffected cells

*Represents female Rett syndrome; **Represents male duplication disorder; WT = wildtype

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Pini G, et al. Orphanet Journal of Rare Diseases (2016) 11:132.

Acts As a Genetic Thermostat, Limiting Transgene Expression

EXACT miRNA controls transgene levels to targeted range

Regulatory elements designed to avoid off- target effects

EXACT is expected to enable gene therapy for Rett syndrome and other complex disorders

ExcessProtein

Transcript Expressed

Destroyed

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Designed to Widen Therapeutic Window and Enable Gene Therapy for Rett Syndrome

Too low

Rett Syndrome

~50% of cells express WT levels of MeCP2

~50% are MeCP2 deficient

NGN-401 designed to

provide therapeutic and tolerable levels of MeCP2 within this window

MeCP2 levels

Therapeutic and tolerable

treatment window

Too high MeCP2 Overexpression Toxicity

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Disclaimer

Neurogene Inc. published this content on March 24, 2025, and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on March 24, 2025 at 20:40:09.743.