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• Sarepta Therapeutics (SRPT) for rare neurological gene therapies.
• Bluebird Bio (BLUE) for severe brain disorders.

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• NGN-101: An investigational gene therapy for CLN5 Batten disease, a rare and fatal inherited neurological disorder.
• NGN-401: A gene therapy candidate being developed for Rett syndrome, a severe neurodevelopmental disorder.
• NGN-201: An investigational gene therapy for ataxia-telangiectasia (A-T), a rare, progressive, neurodegenerative genetic disease.

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Neurogene (NGNE) is a clinical-stage biotechnology company focused on developing gene therapies for rare neurological diseases. As of its current stage, Neurogene has not yet commercialized any products and therefore does not have major commercial customers in the traditional sense, as it does not generate revenue from product sales.

However, should Neurogene's gene therapies receive regulatory approval and become commercially available, its sales model would primarily involve selling to other companies within the healthcare ecosystem. The major categories of these potential customer companies would include:

• Hospitals and Academic Medical Centers: These institutions are typically the sites for the administration of complex gene therapies. They would be the direct purchasers of Neurogene's approved products to treat patients.
• Specialty Pharmacies and Distributors: These companies specialize in the handling, storage, and distribution of high-cost, complex biological drugs like gene therapies. They would facilitate the delivery of products to treatment centers or, in some cases, directly to patients under specific programs, acting as key intermediaries in the supply chain.

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Rachel McMinn, Ph.D., Founder, Chief Executive Officer and Executive Chair of Board
Dr. Rachel McMinn founded Neurogene in January 2018 with the mission of developing genetic medicines for neurologically-impaired people. Before establishing Neurogene, she held the position of Chief Business and Strategy Officer at Intercept Pharmaceuticals, a company focused on liver disease. Prior to her industry career, Dr. McMinn spent 13 years as an award-winning biotechnology analyst at firms such as Bank of America Merrill Lynch, Cowen, and Piper Jaffray. She earned her Ph.D. from The Scripps Research Institute and a B.A. from Cornell University.

Christine Mikail, J.D., President and Chief Financial Officer
Christine Mikail joined Neurogene in 2019 and is responsible for Corporate Strategy and Business Development, Portfolio Management, Operations, and Finance. With over two decades of experience in the biotechnology and pharmaceutical sectors, Ms. Mikail previously served as Chief Administrative Officer and Head of External Business Development/Alliance Management and General Counsel at Axovant Sciences, Inc., where she was instrumental in the company's $362 million IPO. Her career also includes senior executive roles at NPS Pharmaceuticals, Inc., Dendreon Corporation, Eli Lilly and Company, and ImClone Systems. Ms. Mikail began her career as a corporate and securities lawyer. She holds a B.A. from Rutgers University and a J.D. from Fordham University School of Law.

Stuart Cobb, Ph.D., Chief Scientific Officer
Dr. Stuart Cobb is listed as a founding leader and Chief Scientific Officer at Neurogene.

Julie Jordan, M.D., Chief Medical Officer
Dr. Julie Jordan is a founding leader and serves as the Chief Medical Officer at Neurogene. She was hired as Chief Medical Officer, indicating a focus on strengthening the clinical team.

Effie Albanis, M.D., Senior Vice President, Early Clinical and Translational Research
Dr. Effie Albanis is a founding leader and holds the position of Senior Vice President, Early Clinical and Translational Research at Neurogene.

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The key risks to Neurogene (NGNE) are primarily centered around its clinical-stage drug development, regulatory challenges, and associated financial sustainability.

1. Clinical Trial Outcomes and Regulatory Approval for NGN-401: Neurogene's valuation is largely tied to the success of its lead gene therapy program, NGN-401, for Rett syndrome. The program recently faced a serious adverse event (SAE) in the high-dose cohort, leading to the death of a patient and the discontinuation of further enrollment at that dose level. While the FDA has permitted the Phase 1/2 study to continue with the low dose, the inherent uncertainties of clinical trial outcomes, potential for further adverse events, and the rigorous regulatory approval process remain significant hurdles for ultimate commercialization. The company also faces risks related to reporting data on predicted timelines.
2. Discontinuation and Uncertainty of the NGN-101 Program: Neurogene has halted the advancement of its NGN-101 program for CLN5 Batten disease due to challenges in reaching alignment with the FDA on a streamlined registrational pathway. Although the company is exploring alternatives such as partnering or out-licensing, the success of these efforts is uncertain, and Neurogene will continue to incur ongoing costs from post-dosing patient observations in the Phase 1/2 clinical trial without a clear path to commercialization.
3. Financial Health and Need for Capital: As a clinical-stage biotechnology company, Neurogene exhibits typical financial challenges, including negative earnings per share and return on equity. The company also has a significant negative free cash flow, underscoring the necessity for careful cash management as its clinical trials progress. The biotech sector is inherently high-risk and high-reward, and as a pre-revenue company, Neurogene's long-term financial viability is highly dependent on the successful development and commercialization of its product candidates.

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Neurogene (NGNE) is a clinical-stage company focused on developing genetic medicines for rare neurological diseases. As such, its future revenue growth over the next 2-3 years will primarily be driven by the successful advancement and potential commercialization of its pipeline products.

Here are 3-5 expected drivers of Neurogene's future revenue growth:

• **Advancement and potential commercialization of NGN-401 for Rett Syndrome:** NGN-401 is Neurogene's lead gene therapy candidate, for which the company has announced positive regulatory updates and plans to initiate dosing in the Embolden™ registrational trial in the fourth quarter of 2025. The trial is designed to evaluate participants as young as three years of age with a primary endpoint incorporating measures considered clinically meaningful. Successful progression through this trial and eventual regulatory approval would be a significant driver of future revenue. The company expects to report updated clinical efficacy and safety data for NGN-401 in the second half of 2025.
• **Progress of NGN-101 for CLN5 Batten Disease through clinical trials:** Neurogene is also advancing NGN-101 for CLN5 Batten disease, which is currently in Phase 1/2 clinical trials. Positive data and continued progression of this program are crucial for building out the company's pipeline and future revenue streams. Neurogene anticipates sharing data from this program later in 2024.
• **Expansion of the gene therapy pipeline leveraging the proprietary EXACT platform:** Neurogene is actively building a broader gene therapy pipeline to address significant unmet needs in rare neurological diseases. Its proprietary EXACT gene regulation platform technology, designed to deliver precise transgene expression and avoid potential toxicities, positions the company for potential breakthroughs that could drive future profitability. The company plans to advance at least one product from its discovery-stage pipeline into the clinic in 2025.
• **Successful market entry and adoption of approved genetic medicines for rare neurological diseases:** As a clinical-stage company, Neurogene's long-term revenue growth depends on its ability to bring innovative genetic medicines to market. Its strategic focus on rare neurological diseases aims to address areas of high unmet medical need, which, upon successful development and regulatory approval, could lead to significant market penetration and revenue generation.

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Share Issuance

• In November 2024, Neurogene announced an agreement for a Private Investment in Public Equity (PIPE) financing, which was expected to generate approximately $200 million in gross proceeds and close around November 5, 2024.
• This PIPE financing was projected to result in approximately 21.0 million shares of common stock outstanding.
• The capital raised from the PIPE financing is intended to extend Neurogene's financial runway into the second half of 2027.

Inbound Investments

• In November 2024, Neurogene secured approximately $200 million through an oversubscribed PIPE financing, with participation from prominent healthcare investment firms including RTW Investments and Casdin Capital.

Capital Expenditures

• Neurogene's capital expenditures for the latest twelve months were $1.211 million.
• Capital expenditures peaked in December 2021 at $18.369 million.
• The company has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas, which conducts GMP production of NGN-401 and supports pivotal clinical development activities.