Neurogene Inc. · NASDAQ:NGNE
Neurogene — Investor Presentation (January 2026)
28 slides from Neurogene's investor presentation, Jan 12, 2026
Slide 1: Every Breakthrough Begins With Belief – Corporate Presentation
EVERY BREAKTHROUGH BEGINS WITH BELIEF January 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 for
Slide 5: 2025 Accomplishments Set Up for Pivotal Year in 2026
2025 Accomplishments Set Up for Pivotal Year in 2026 5 Completed enrollment of Phase 1/2 trial Obtained FDA alignment on Embolden registrational trial Dosed multiple participants in Embolden Reported positive interim Phase 1/2 data Received PRIME designation from EMA, the fifth special regulatory de
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 Best-in-Class Gene Therapy for Rett Syndrome
8 NGN-401: Purposefully Designed to Be the Best-in-Class Gene Therapy for Rett Syndrome ICV = Intracerebroventricular 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 Syste
Slide 9: NGN-401 Phase 1/2 Trial
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Slide 10: Dosing Completed in NGN-401 Phase 1/2 Clinical Trial in Females with Rett Syndrome
10 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 11: Baseline Characteristics of the Pediatric Participants in Phase 1/2
Baseline Characteristics of the Pediatric Participants in Phase 1/2 11 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 12: NGN-401 Drove Clinically Meaningful and Durable Improvement Across Key Rett Domains
NGN-401 Drove Clinically Meaningful and Durable Improvement Across Key Rett Syndrome Domains 12As 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 13: Caregivers Seek Treatment that Improves Daily Living and Leads to Durable Improvements
13 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 14: NGN-401 Drove Durable Accumulation of Multidomain Milestones
14 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 15: Embolden Registrational Trial Overview
EMBOLDEN REGISTRATIONAL TRIAL OVERVIEW
Slide 16: Alignment with FDA on Single Registrational Trial to Support BLA Submission
Developmental Milestones • CGI-I score of ≤ 2 • Gain from baseline of at least 2 developmental milestones 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
Slide 17: 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 17 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 18: Responders in Phase 1/2 Trial Exceed the Bar for Success in Embolden
18 35% (7 of 20) Pre-Specified Minimum Response Rate for Success 80% Response Rate (4 of 5) % R e s p o n d e rs a t 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
Slide 19: Building a Strong Foundation for Commercialization
19 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 20: NGN-401 Positioned for Leadership in Rett Syndrome with 2026 Catalysts
Compelling Interim Clinical Data: Multidomain, durable improvements across spectrum of disease severity 20 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 21: Appendix: Interim Phase 1/2 Data Update Participant Vignettes and Safety Overview
APPENDIX: INTERIM PHASE 1/2 DATA UPDATE PARTICIPANT VIGNETTES AND SAFETY OVERVIEW
Slide 22: Pt:1 Gained Developmental Milestones Across All Core Domains Out to 24 Months
22 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 23: Pt:2 Gained Developmental Milestones Across All Core Domains Out to 18 Months
23 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 24: Pt:3 Gained Developmental Milestones Across All Core Domains Out to 18 Months
24 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 25: Pt:4 Gained Developmental Milestones Across Core Domains Out to 15 Months
25 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 26: Pt:7 Gained Developmental Milestone at Early Timepoint
26 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 27: Pt:8 Gained Developmental Milestones Across Core Domains at Early Timepoint
27 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
Slide 28: NGN-401 Remains Generally Well Tolerated at the 1E15 vg Dose Level
28 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