Board Change • May 21
High number of new directors There are 5 new directors who have joined the board in the last 3 years. President, CFO & Director Christine Cvijic was the last director to join the board, commencing their role in 2026. The company’s lack of board continuity is considered a risk according to the Simply Wall St Risk Model. Announcement • May 12
Neurogene Inc Highlights Data Supporting Therapeutic Rationale For ICV Delivery Method In CNS-Targeted Gene Therapy Neurogene Inc. highlighted data at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting supporting the intracerebroventricular (ICV) route of administration as a routine approach for delivering medicines, including gene therapies, to treat central nervous system (CNS) disorders. The presentation further highlights that the ICV procedure is routine, and administration takes approximately 10 minutes; therefore, the clinical safety and monitoring in gene therapy programs is driven primarily by factors related to transgene expression, dose and total viral load rather than the delivery procedure itself. The poster, “Gene Therapy Targeting CNS Diseases: ICV Administration as a Growing Standard for Delivery,” provides an overview of ICV delivery of NGN-401 gene therapy for Rett syndrome with findings from a preclinical study as well as an ongoing Phase 1/2 trial in participants with Rett syndrome, which has completed dosing. The ICV procedure is utilized by pediatric and adult neurosurgeons tens of thousands of times annually, and the administration of NGN-401 gene therapy takes approximately 10 minutes. Compared to intrathecal lumbar (IT-L) delivery of NGN-401, ICV delivery showed greater biodistribution to the key areas of the brain, including the motor cortex, frontal cortex and cerebellum, and CNS that underlie Rett syndrome in preclinical models. In the Phase 1/2 trial of NGN-401, all participants who received the 1E15 vg dose in the pediatric cohort (ages 4-10; n=8) showed functional improvements, with a total of 35 developmental milestones gained with no plateau up to 24 months, and NGN-401 and the ICV procedure were generally well-tolerated as of the data cutoff date of October 30, 2025 (n=10). NGN-401 is also being administered through ICV in the Embolden registrational clinical trial, and investigators have the option to discharge participants one day after the procedure and implement an outpatient gene therapy monitoring protocol. Clinical safety in CNS gene therapy is driven primarily by vector biology, dose and transgene expression rather than the route of administration itself. NGN-401 is an investigational AAV9 gene therapy in late-stage clinical development as a potential best-in-class, one-time treatment for Rett syndrome. It is the only clinical candidate to deliver the full-length human MECP2 gene and includes Neurogene’s EXACT transgene regulation technology, which is designed to deliver consistent, tightly controlled MeCP2 protein expression on a cell-by-cell basis. NGN-401 is delivered through intracerebroventricular administration to achieve the broadest targeting directly to the brain and nervous system based on nonclinical biodistribution data. NGN-401 is being evaluated in the Embolden registrational clinical trial. Interim data from the Phase 1/2 trial (as of October 30, 2025) have shown that participants experienced multidomain, durable gains with continued skill acquisition observed over time, and NGN-401 at the 1E15 vg dose has been generally well-tolerated. NGN-401 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations and selection for the START Pilot Program from the U.S. Food and Drug Administration, Advanced Therapy Medicinal Product, Orphan and Priority Medicines designations from the European Medicines Agency and Innovative Licensing and Application Pathway designation from the United Kingdom Medicines and Healthcare products Regulatory Agency. Announcement • Apr 23
Neurogene Inc. Announces Executive and Board Appointments Neurogene Inc. announced the appointment of Christy Shafer as Chief Commercial Officer and the appointment of Christine Mikail, J.D., to its Board of Directors. Ms. Shafer brings more than 20 years of industry experience building and leading high-performing commercial organizations and launching therapies for rare neurological diseases. Ms. Mikail brings to the Board deep expertise in strategic transactions, capital formation, and corporate development, with a track record of building and positioning companies for long-term value creation. Since joining Neurogene, Ms. Mikail has played a central role in shaping the Company's long-term strategy, strengthening its financial foundation, and supporting advancement of its clinical and corporate objectives. Christy Shafer most recently served as Senior Vice President and General Manager, North America, of Avidity Biosciences, a late-stage biotech developing RNA therapeutics for rare neuromuscular diseases that was acquired by Novartis. She was responsible for building and leading Avidity's North America commercial organization in advance of its first U.S. product launches. Prior to Avidity, Ms. Shafer served as Chief Commercial Officer at Marinus Pharmaceuticals, where she built and led the commercial organization and launched Ztalmy®, the first approved therapy for seizures associated with CDKL5 deficiency disorder, which led to Marinus Pharmaceuticals' acquisition by Immedica Pharma. Previously, Ms. Shafer held leadership roles of increasing responsibility at Alexion Pharmaceuticals, where she supported multiple neurology launches, including Soliris® for generalized myasthenia gravis and neuromyelitis optica spectrum disorder, as well as earlier commercial leadership positions at Pacira Pharmaceuticals and Sanofi Biosurgery (formerly Genzyme). She holds a Bachelor of Life Science degree in Cell and Molecular Biology and Genetics from the University of Maryland, College Park, and completed postbaccalaureate studies in immunology and pharmacology. 
