Neurogene Inc. · NASDAQ:NGNE
Neurogene — Investor Presentation (March 2026)
33 slides from Neurogene's investor presentation, Mar 24, 2026
Slide 1: Every Breakthrough Begins With Belief – March 2026 Corporate Presentation
EVERY BREAKTHROUGH BEGINS WITH BELIEF March 2026 Corporate Presentation
Slide 2: Forward Looking Statements
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 conditi
Slide 3: Our Mission and Approach
3 To develop life-changing genetic medicines for people and their families impacted by devastating neurological diseases OUR MISSION Biology-first design Precision delivery for maximum drug distribution EXACT platform for precise transgene expression Driven by patients and families in need OUR APPRO
Slide 4: NGN-401 Rapidly Advancing Towards Commercialization for Rett Syndrome
NGN-401 is Rapidly Advancing Towards Commercialization as a Potential Transformative One-Time Treatment for Rett Syndrome 4 Clear Path to Registration with Single Registrational Trial for Broadest Age Range • Key elements of Embolden registrational trial aligned with FDA, including a single trial fo
Slide 5: NGN-401 for Rett Syndrome
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Slide 6: Rett Syndrome: Rare, Debilitating, Progressive, Neurodevelopmental Disorder
6 Cause: Variants in the MECP2 gene on the X chromosome lead to deficiency of functional MeCP2 protein MeCP2 is a DNA-binding protein essential for normal brain and nervous system function Onset: Developmental delay occurs at 6-18 months, followed by loss of previously acquired milestones during reg
Slide 7: Multi-Billion-Dollar Market Opportunity for Disease-Modifying Gene Therapy for Rett Syndrome
7 Multi-Billion-Dollar Market Opportunity for Disease-Modifying Gene Therapy for Rett Syndrome 1 Major market prevalence based on internal estimates; U.S. prevalence estimate based on published incidence rates; Laurvick CL, et al. J Pediatr 2006;148(3):347–35. 2 WW incidence estimate based on publis
Slide 8: NGN-401: Purposefully Designed to Be the Best-in-Class Gene Therapy for Rett Syndrome
8 NGN-401: Purposefully Designed to Be the Best-in-Class Gene Therapy for Rett Syndrome Full-length MECP2 to translate functional MeCP2 protein NGN-401 Construct Only Gene Therapy Designed to Safely Deliver Full-length Protein to Key Areas of the Brain and Nervous System to Maximize Benefit ICV deli
Slide 9: ICV Delivery Achieves Greater Expression in the Brain Compared to IT-L
9 ICV Delivery Achieves Greater Expression in the Brain Compared to IT-L ICV = Intracerebroventricular IT-L = intrathecal lumbar ESGCT 2025 ICV route of delivery has been shown in preclinical models to have greater targeting of brain and nervous system regions underlying Rett syndrome pathophysiolog
Slide 10: NGN-401 Phase 1/2 Trial
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Slide 11: Dosing Completed in NGN-401 Phase 1/2 Clinical Trial in Females with Rett Syndrome
11 Dosing Completed in NGN-401 Phase 1/2 Clinical Trial in Females with Rett Syndrome Trial evaluating 1E15 vg dose of NGN-401 n=8 Ages 4-10 n=2 Ages > 11 Open-label, multicenter trial designed to assess the safety, tolerability and efficacy of NGN-401 Key Eligibility Criteria: Females with Classic
Slide 12: Baseline Characteristics of the Pediatric Participants in Phase 1/2
Baseline Characteristics of the Pediatric Participants in Phase 1/2 12 Pediatric Cohort (n=8) Pt:1 Pt:2 Pt:3 Pt:4 Pt:5 Pt:6 Pt:7 Pt:8 Age at Dosing (Years) 7 4 6 7 6 4 6 8 Baseline CGI-S Score 4 Moderately Ill 5 Markedly Ill 5 Markedly Ill 5 Markedly Ill 5 Markedly Ill 5 Markedly Ill 4 Moderately Il
Slide 13: NGN-401 Drove Clinically Meaningful and Durable Improvement Across Key Rett Syndrome Domains
NGN-401 Drove Clinically Meaningful and Durable Improvement Across Key Rett Syndrome Domains 13As of data cutoff date of October 30, 2025; Neurogene press release (November 12, 2025) 100% showed functional improvements across core disease domains – fine motor/hand function, gross motor/ambulation an
Slide 14: Caregivers Seek Treatment that Improves Daily Living and Leads to Durable Improvements
14 Caregivers Seek Treatment that Improves Daily Living and Leads to Durable, Multidomain Improvements Fine Motor/Hand Function Improvements Enables self-feeding, quicker meals, family/social dining and independence E.g., Transferring/grasping objects, finger feeding, drinking from a cup, using uten
Slide 15: NGN-401 Drove Durable Accumulation of Multidomain Milestones
15 NGN-401 Drove Durable Accumulation of Multidomain Milestones Across Core Domains That Matter Most to Caregivers 3 4 5 7 92 6 4 7 8 4 8 12 12 14 3 Mos. 6 Mos. 9 Mos. 12 Mos. >12 Mos. Interim Phase 1/2 Data Showed Developmental Milestones Increased by Domain Over Time Post-NGN-401 Treatment n=8 n=8
Slide 16: NGN-401 Delivers Durable, Multidomain Improvements that Translate to Real Life Impact
16 NGN-401 Delivers Durable, Multidomain Improvements that Translate to Real Life Impact Can communicate needs & wants, and foster greater social connections Says words with meaning Follows daily routines, e.g., getting ready for school Communicates needs and wants; can ID colors Engages with friend
Slide 17: Phase 1/2 Caregiver Testimonials Following Treatment with NGN-401
17 Phase 1/2 Caregiver Testimonials Following Treatment with NGN-401 Highlight Improvements Important to Families As of data cutoff date of October 30, 2025 “Sleep improved significantly as she is able to sleep all through the night without any issue.” “Her understanding has improved, especially wit
Slide 18: NGN-401 Remains Generally Well Tolerated at the 1E15 vg Dose Level
18 NGN-401 Remains Generally Well Tolerated at the1E15 vg Dose Level As of data cutoff date of October 30, 2025 TEAE = Treatment emergent adverse event • All TEAEs related to NGN-401 have been Grade 1 (mild) or Grade 2 (moderate) in severity; the majority are known potential risks of AAV and have re
Slide 19: Embolden Registrational Trial Overview
EMBOLDEN REGISTRATIONAL TRIAL OVERVIEW
Slide 20: Alignment with FDA on Single Registrational Trial to Support BLA Submission
20 Alignment with FDA on Single Registrational Trial to Support BLA Submission; Enrollment Underway in Embolden Trial Immunosuppression: Steroid prophylaxis during first 90 days, followed by taper Developmental Milestones • CGI-I score of ≤ 2 • Gain from baseline of at least 2 developmental mileston
Slide 21: Pre-Specified Developmental Milestones Aligned with FDA, KOLs and Caregivers
28 Pre-Specified Developmental Milestones From Natural History Database Aligned with FDA, KOLs and Caregivers as Meaningful 21 Fine Motor/ Hand Function Gross Motor/Ambulation Communication 1.2% - 3% 0.3 – 6.6% Reached for toy Taken a drink from a cup held without assistance Used raking grasp to ret
Slide 22: Responders in Phase 1/2 Trial Exceed the Bar for Success in Embolden
22 35% (7 of 20) Pre-Specified Minimum Response Rate for Success 80% Response Rate (4 of 5) % R es p o n d er s at 1 2 m o n th s Phase 1/2 Responders in Phase 1/2 Trial Exceed the Bar for Success Established as the Primary Endpoint in the Embolden Trial As of data cutoff date of October 30, 2025 Ph
Slide 23: Commercial Readiness & Key Anticipated Milestones
COMMERCIAL READINESS & KEY ANTICIPATED MILESTONES
Slide 24: Building a Strong Foundation for Commercialization
24 Building a Strong Foundation for Commercialization Internal CMC capabilities and manufacturing facility to produce commercial product Positioning Embolden clinical trial sites for rapid conversion to commercial sites at launch Payor research confirms strong reimbursement potential for NGN-401 Emb
Slide 25: Wholly Owned and Fully Integrated In-House AAV Manufacturing
Wholly Owned and Fully Integrated In-House AAV Manufacturing 25 GMP Manufacturing Toxicology Batch Manufacturing Process Development Quality Assurance Quality Control Analytical Development Flexibility to manufacture AAV product at low cost Own product quality and development timelines Process devel
Slide 26: NGN-401 Positioned for Leadership in Rett Syndrome with Multiple Catalysts
Compelling Interim Clinical Data: Multidomain, durable improvements across spectrum of disease severity 26 Single Registrational Trial Enrolling: Enables approval for broad population NGN-401 is Positioned for Leadership in Rett Syndrome with Multiple Value-Creating Catalysts Expected in 2026 and Be
Slide 27: Appendix: Interim Phase 1/2 Data Update Participant Vignettes
APPENDIX: INTERIM PHASE 1/2 DATA UPDATE PARTICIPANT VIGNETTES
Slide 28: Pt:1 Gained Developmental Milestones Across All Core Domains with Durability out to 24 Months
28 Pt:1 Gained 11 Developmental Milestones Across All Core Domains with Durability out to 24 Months Walking, ataxic gait, no ability to climb stairs Raking, no ability to hold objects Severe impairment, unable to follow commands, indicate wishes Pt:1 Developmental Milestones Post-NGN-401 Months Post
Slide 29: Pt:2 Gained Developmental Milestones Across All Core Domains with Durability out to 18 Months
29 Pt:2 Gained 10 Developmental Milestones Across All Core Domains with Durability out to 18 Months As of data cutoff date of October 30, 2025 *Not included in Embolden developmental milestones list; previously reported developmental milestones from other validated scales Images are representative o
Slide 30: Pt:3 Gained Developmental Milestones Across All Core Domains with Durability out to 18 Months
30 Pt:3 Gained 6 Developmental Milestones Across All Core Domains with Durability out to 18 Months As of data cutoff date of October 30, 2025 Images are representative of developmental milestones Pt:3 Baseline: 6 Yrs Old Cannot sit, stand or walk independently Raking grasp Post Treatment with NGN-40
Slide 31: Pt:4 Gained Developmental Milestones Across Core Domains with Durability out to 15 Months
31 Pt:4 Gained 4 Developmental Milestones Across 2 Core Domains with Durability out to 15 Months Pt:4 Baseline: 7 Yrs Old Raking grasp, unable to hold objects Post Treatment with NGN-401 Unable to follow commands Pt:4 Developmental Milestones Post-NGN-401 Months Post-NGN-401 3 6 9 12 15 Fine Motor/H
Slide 32: Pt:7 Gained Developmental Milestone at Early Timepoint
32 Pt:7 Gained 1 Developmental Milestone at Early Timepoint Pt:7 Developmental Milestones Post-NGN-401 Months Post-NGN-401 3 6 Fine Motor/Hand Function Used a spoon/fork to eat without assistance Pt:7 Baseline: 6 Yrs Old Post Treatment with NGN-401 Could not use utensils As of data cutoff date of
Slide 33: Pt:8 Gained Developmental Milestones Across Core Domains at Early Timepoint
33 Pt:8 Gained 3 Developmental Milestones Across 2 Core Domains at Early Timepoint Pt:8 Baseline: 8 Yrs Old Unable to bend at waist Pt:8 Developmental Milestones Post-NGN-401 Months Post-NGN-401 3 6 Gross Motor/ Ambulation Bent down, touched floor, and recovered* Communication Followed command w