Neurogene : Corporate Presentation June 2024

Corporate Presentation

June 2024

Disclaimer

Forward Looking Statements

This communication contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current expectations and beliefs of the management of Neurogene, as well as assumptions made by, and information currently available to, management of Neurogene, including, but not limited to, statements regarding the therapeutic potential and utility, efficacy and clinical benefits of its programs, including its EXACTTM technology, NGN-401 and NGN-101; market opportunities for Neurogene's product candidates; the safety and tolerability profile of NGN-401 and NGN-101; trial designs, clinical development plans and timing for NGN-401 and NGN-101, including enrollment and dosing in both cohorts of the NGN- 401 Phase 1/2 clincal trial for Rett Syndrome, anticipated clinical data results in NGN-401 Phase 1/2 trial for Rett syndrome and anticipated clinical data results in NGN-101 Phase 1/2 trial for CLN5 Batten disease; anticipated impact of expansion of the Phase 1/2 trial for NGN-401, including the addition of a high dose Cohort 2 and expansion of the clinical trial into the United Kingdom and Australia; future interactions with U.S. or foreign regulatory authorities, including participation the Federal Drug Administration's START program; anticipated early-stage discovery and expectations regarding the initiation of future clinical trials for programs in development; and Neurogene's cash runway. Forward- looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as "may," "will," "should," "would," "expect," "anticipate," "plan," "likely," "believe," "estimate," "project," "intend," and other similar expressions or the negative or plural of these words, or other similar expressions that are predictions or indicate future events or prospects, although not all forward-looking statements contain these words. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: Neurogene's limited operating history; the significant net losses incurred since inception of Neurogene; the ability to raise additional capital to finance operations; the ability to advance product candidates through non-clinical and clinical development; the ability to obtain regulatory approval for, and ultimately commercialize, Neurogene's product candidates; Neurogene's limited experience in designing and conducting clinical trials; the ability to identify and pivot to other programs, product candidates, or indications that may be more profitable or successful than Neurogene's current product candidates; expectations regarding the market and potential for Neurogene's current product candidates; expectations regarding the potential tolerability, safety or efficacy for Neurogene's current product candidates; the ability to attract, hire, and retain skilled executive officers and employees; reliance on third parties, contract manufacturers, and contract research organizations; the ability of Neurogene to protect its intellectual property and proprietary technologies; risks related to Neurogene's ability to correctly estimate its respective operating expenses, including its projected cash runway; and legislative, regulatory, political and economic developments and general market conditions.

The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company's most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commission (SEC), as well as risk factors associated with companies, such as Neurogene, that operate in the biopharma industry. These forward-looking statements involve a number of risks, uncertainties (some of which are beyond Neurogene's control) or other assumptions that may cause actual results or performance to be materially different from those expressed or implied by these forward-looking statements. Nothing in this communication should be regarded as a representation by any person that the forward- looking statements set forth herein will be achieved or that the contemplated results of any such forward-looking statements will be achieved. Forward-looking statements in this communication speak only as of the day they are made and are qualified in their entirety by reference to the cautionary statements herein. Except as required by applicable law, Neurogene undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Industry and Market Data

Certain information contained in this Presentation relates to or is based on studies, publications, surveys and Neurogene's own internal estimates and research. In this Presentation, Neurogene relies on, and refers to, publicly available information and statistics regarding market participants in the sector in which Neurogene competes and other industry data. Any comparison of Neurogene to any other entity assumes the reliability of the information available to Neurogene. Neurogene obtained this information and statistics from third-party sources, including reports by market research firms and company filings. In addition, all of the market data included in this Presentation involve a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. Finally, while Neurogene believes its internal research is reliable, such research has not been verified by any independent source and Neurogene has not independently verified the information.

Trademarks

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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:26 cash runway enables operations beyond clinical inflection points

EXACT: Expression Attenuation via Construct Tuning	3

Neurogene Clinical Stage Pipeline

Transgene Regulation CNS + Ocular Delivery

Product	Indication	IND* Enabling	Phase I/2	Pivotal	Near-Term
Candidate	Expected Milestones
NGN-401	Rett Syndrome				Interim Data
			4Q:24,
					Additional Data 2H:25
NGN-101	CLN5 Batten Disease				Interim Data
			2H:24

*IND = investigational new drug.

Multiple discovery stage assets in development with plans to advance one

program into the clinic in 2025

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Developed to Solve the Limitations of Conventional Gene Therapy in Complex Neurological Disorders

Today's Gene Therapy is Limited By:

Variable Gene Expression

Safety Limitations

Neurogene's

Solutions:

Novel, modular EXACT gene regulation technology and other regulatory elements designed to optimize transgene expression to maximize the therapeutic window

Novel and proprietary EXACT gene regulation technology designed to avoid transgene related toxicity associated with conventional gene therapy

Inefficient Gene Delivery

Select ICV delivery approach to maximize AAV9 distribution to target CNS tissues

Design products to maximize potency and purity for potentially optimized efficacy/safety profile

ICV = intracerebroventricular

AAV = adeno-associated virus5 CNS = central nervous system

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

• 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
• Flexibility to rapidly adapt CMC execution to program needs

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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Experienced Leadership Team

Management Team

Rachel McMinn, Ph.D.	Christine Mikail, J.D.	Julie Jordan, M.D.	Stuart Cobb, Ph.D.
Founder and CEO	President and CFO	CMO	CSO

Ricardo Jimenez	Effie Albanis, M.D.	Andrew Mulberg, M.D.	Arvind Sreedharan
SVP, Technical Operations	SVP, Early Clinical and	SVP, Regulatory Affairs	SVP, Business Operations
	Translationall Research

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NGN-401 for Rett Syndrome

Leveraging EXACT gene regulation technology

Rett Syndrome - Devastating Disorder with High Unmet Need

Genetics

• X-Linkeddisorder causing mutations in the gene encoding for methyl-CpG binding protein 2 (MeCP2)
• Unknown incidence in boys, but typically lethal by ~3 years of age due to no healthy copy of MeCP2

Compelling Market Opportunity

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

• WW incidence - 1:10,000 females

High Unmet Need

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

• Significant unmet need remains for new treatment options

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

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

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Rett Syndrome Treatment Requires Tight Transgene Regulation

Rett Syndrome*					MeCP2
					Duplication Disorder**

Too little	Balanced	Too much
gene expression drives disease	treatment goal	gene expression drives disease

~50% of cells express WT levels of MeCP2		~100% cells express 2x MeCP2 levels
~50% are MeCP2 deficient

• Rett syndrome (RTT) is a severe neurological disorder caused by mosaic mutations in X-linked MeCP2 gene
• Mice modeling RTT recapitulate many neurological phenotypes observed clinically; disease reversibility has been demonstrated in both immature and mature adult animals

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	10
Pini G, et al. Orphanet Journal of Rare Diseases (2016) 11:132.