Neurogene : Corporate Presentation June 2025

Published: 2025-06-09 08:54:04 ET NGNE

Published on 06/09/2025 at 08:54

June 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

Neurogene Clinical Stage Pipeline

Product

Candidate

Indication

IND* Enabling

Phase I/2

Pivotal

Near-Term

Expected Milestones

NGN-401

Rett Syndrome

NGN-101

CLN5 Batten Disease

Evaluating Opportunities for

Program

Registrational Trial Plans Update

1H:25

Additional Interim Data 2H:25

*IND = investigational new drug

Wholly Owned and Fully Integrated In-House AAV Manufacturing

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

Flexibility to manufacture AAV product at low cost

Own product quality and development timelines

Process development expertise supports both HEK293 and Sf9/rBV manufacturing platforms

Current research and clinical-grade manufacturing capabilities are designed for

commercial-grade product to avoid potential future comparability challenges

Flexibility to rapidly adapt CMC execution to program needs

Disclaimer

Neurogene Inc. published this content on June 08, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on June 09, 2025 at 12:53 UTC.