Neurogene Inc. · NASDAQ:NGNE
Neurogene — Investor Presentation (August 2026)
37 slides from Neurogene's investor presentation, August 2026
Slide 1: Every Breakthrough Begins with Belief
EVERY BREAKTHROUGH BEGINS WITH BELIEF Corporate Presentation August 2026
Slide 2: Disclaimer
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 financ
Slide 3: Our Mission and Approach
OUR MISSION To develop life-changing genetic medicines for people and their families impacted by devastating neurological diseases OUR APPROACH Biology-first Precision delivery for EXACT platform for Driven by patients and design maximum drug precise transgene families in need distribution expressio
Slide 4: NGN-401 is Rapidly Advancing Towards Commercialization
NGN-401 is Rapidly Advancing Towards Commercialization as a Potential Transformative One-Time Treatment for Rett Syndrome Potential Best-in-Class Treatment for Rett Syndrome • Compelling clinical evidence showing durable multidomain improvements demonstrated across full spectrum of disease severity
Slide 5: NGN-401 for Rett Syndrome
ad YNevrocene NGN-401 FOR RETT SYNDROME ¢¢¢
Slide 6: No Approved Disease-Modifying Therapies for Rett Syndrome
No Approved Disease-Modifying Therapies for Rett Syndrome NGN-401 Gene Therapy Targets Root Cause The Unmet Need The Market The Opportunity • Developmental regression begins at • The Rett syndrome community is 6–18 months 15,000–20,000 increasingly seeking therapies that patients — US, EU & UK can d
Slide 7: NGN-401: Purposefully Designed to Be the Best-in-Class Gene Therapy
NGN-401: Purposefully Designed to Be the Best-in-Class Gene Therapy for Rett Syndrome NGN-401 Construct Full-length MECP2 to translate functional MeCP2 protein EXACT transgene regulation technology designed to safely achieve target levels ICV delivery to achieve the broadest targeting of brain and n
Slide 8: NGN-401 Clinical Program
AY YNevrocene NGN-401 CLINICAL PROGRAM ¢¢¢
Slide 9: NGN-401 Phase 1/2 Trial Update
NGN-401 Phase 1/2 Trial Update: Clinically Meaningful, Durable Improvement Across Key Rett Syndrome Domains, Deepens Over Time 100% of Phase 1/2 participants (N=10) gained ≥1 47 total milestones gained 30 developmental milestone and improved on CGI-I months of continued improvement post-dose 80% met
Slide 10: Long-term Phase 1/2 Data Demonstrate Broad, Consistent Multidomain Gains
Long-term Phase 1/2 Data Demonstrate Broad, Consistent Multidomain Developmental Gains that Support Registration Regardless of Age, Disease Severity or Genotype 100% of Phase 1/2 participants (N=10) gained ≥1 developmental milestone and improved on CGI-I at ≥12 months Rapid onset of clinical respons
Slide 11: NGN-401 Clinical Study Design for Treatment of Rett Syndrome
NGN-401 Clinical Study Design for Treatment of Rett Syndrome Phase 1/2 converted into a single registrational study Study Design • Baseline-controlled, open-label, multicenter, single- Embolden arm pivotal trial (13 US sites) Phase 1/2 Trial Registrational Trial • Evaluates efficacy, safety & tolera
Slide 12: Clearly Defined Developmental Milestones
28 Clearly Defined Developmental Milestones: Derived from Natural History Study & Key Component of Primary Endpoint Fine Motor/ Gross Motor/Ambulation Communication Hand Function Reached for toy Sat with support when placed Responded to familiar Taken a drink from a cup held Sat without support when
Slide 13: Rigorous Embolden Milestone Criteria Evaluation Applied to Phase 1/2 Data
Rigorous Embolden Milestone Criteria Evaluation Applied to Phase 1/2 Data Utilizing the same standardized assessment criteria enables the Phase 1/2 developmental milestone data to be comparable to the Embolden definition Baseline Assessment Post-Treatment Evidence Evaluation Methods Milestones confi
Slide 14: Evaluating the Impact of NGN-401 Across Disease Severity and Ages
Evaluating the Impact of NGN-401 Across Full Spectrum of Disease Severity and Ages Baseline Characteristics of the Phase 1/2 Participants Adult / Adolescent Pediatric Cohort (N=8) Cohort (N=2) Pt:1 Pt:2 Pt:3 Pt:4 Pt:5 Pt:6 Pt:7 Pt:8 Pt:9 Pt:10 Age at Dosing 7 4 6 7 6 4 6 8 18 14 (Years) Baseline 4 5
Slide 15: Restarting Developmental Progression is the Goal
Restarting Developmental Progression is the Goal for a One-time Gene Therapy Restarted Developmental Typical Development Rett Syndrome Progression Organized, cumulative acquisition of Milestones lost in regression, NGN-401 has potential to restart the developmental milestones exceedingly rare to gai
Slide 16: Pt:1 Developmental Milestones Gained
Pt:1 Developmental Milestones Gained in Ordered, Developmental Progression Baseline NGN-401 Restarted Developmental Progression Age: 7 Used a spoon/fork to eat Transferred an object from one Taken a drink from a cup held Used a pincer without assistance hand to the other without assistance grasp Fin
Slide 17: Pt:1 Multidomain Improvements Led to New Abilities
Pt:1 Multidomain Improvements Led to New Abilities Beyond Developmental Milestones At baseline Impaired hand use, Gross motor impairment, Severe communication impairment, (Age 7): raking, no ability to hold objects ataxic, unable to climb stairs unable to follow commands Moves more Joins family Navi
Slide 18: Pt:2 Developmental Milestones Gained
Pt:2 Developmental Milestones Gained in Ordered, Developmental Progression Baseline NGN-401 Restarted Developmental Progression Age: 4 Finger fed Used raking grasp to Reached for Taken a drink from a retrieve an object toy cup held without Fine motor assistance Severe impairment, unable to use hands
Slide 19: Pt:2 Multidomain Improvements Led to New Abilities
Pt:2 Multidomain Improvements Led to New Abilities Beyond Developmental Milestones At baseline Severe impairment, Gross motor impairment, Severe communication impairment, (Age 4): unable to use hands ataxic, needs help to stand unable to follow commands Less caregiver Moves more independently Connec
Slide 20: Pt:3 Multidomain Improvements Led to New Abilities
Pt:3 Multidomain Improvements Led to New Abilities Beyond Developmental Milestones At baseline Impaired hand use, Severe gross motor impairment, Severe communication impairment, (Age 6): raking grasp cannot sit/stand/walk independently unable to follow commands Uses both hands to follow Stands and m
Slide 21: Pt:4 Multidomain Improvements Led to New Abilities
Pt:4 Multidomain Improvements Led to New Abilities Beyond Developmental Milestones At baseline Impaired hand use, Severe gross motor impairment, Severe communication impairment, (Age 7): raking grasp cannot sit/stand/walk independently unable to follow commands Eats on her Sits up by Controls her Pl
Slide 22: Rapid Response Observed Post NGN-401 that Deepened Over Time
Rapid Response Observed Post NGN-401 that Deepened Over Time Response Over Time in the Phase 1/2 Trial Pt:1 Pt:2 Pt:3 Pt:4 47 Total developmental milestones Pt:5 Pt:6 4.7 Average milestones per participant Pt:7 Pt:8 2 months Median time to first response Pt:9 Pt:10 0 3 6 9 12 15 18 24 30 36 Months p
Slide 23: NGN-401 Drove Durable Accumulation of Multidomain Milestones
NGN-401 Drove Durable Accumulation of Multidomain Milestones Across Core Domains That Matter Most to Caregivers Developmental Milestones Increased by Domain Over Time Post-NGN-401 Treatment Fine Motor/Hand Function Expressive & Receptive Communication Gross Motor/Ambulation 47 # of Developmental Mil
Slide 24: Milestones Gained from Embolden List Across Phase 1/2 Participants
Milestones Gained from Embolden List Across 10 Phase 1/2 Participants Treated with NGN-401 21 of 28 Total Milestones Gained from Embolden Milestone List 7/7 7/13 7/8 Fine Motor / Hand Function Gross Motor / Ambulation Communication Reached for toy Sat without support when placed Responded to familia
Slide 25: NGN-401 Remains Generally Well-Tolerated at 1E15 vg Dose
NGN-401 Remains Generally Well-Tolerated at the 1E15 vg Dose Level in the Phase 1/2 Trial • All TEAEs related to NGN-401 have been Grade 1 (mild) or Phase 1/2 Trial 1E15 vg Dose Grade 2 (moderate) in severity; the majority are known Total N = 10 potential risks of AAV and have resolved or are resolv
Slide 26: Robust, Clinically Meaningful Responses Across Participants
Robust, Clinically Meaningful Responses Across Participants Support Potential Market Leading Therapy for Rett Syndrome NGN-401 Phase 1/2 Data (N=10) % of participants gained ≥1 developmental milestone at ≥12 months 100% Average milestones gained per participant 4.7 Total participants achieving: - ≥2
Slide 27: Dosing Completed in Embolden Registrational Trial
Dosing Completed in Embolden Registrational Trial to Support Planned BLA Submission Primary Endpoint at 12 Months Responder-based composite endpoint defined as: • CGI-I of ≤ 3 and • Gain from baseline of any one developmental milestone 33% response rate, or 8 of 24 participants, needed for Single-Ar
Slide 28: Natural History Study Cumulative Incidence Model
Natural History Study Cumulative Incidence Model Shows Beyond Age 3, Milestone Gains Are Rare with Minimal Difference at Age 6 Likelihood of gaining a milestone at > 3 years of age is extremely low, based on RNHS analysis Fine Motor Gross Motor Communication Example Milestone: Taken a drink from a c
Slide 29: Interim Phase 1/2 Data and Trial Design Elements De-risk Embolden
Interim Phase 1/2 Data and Key Trial Design Elements Carried Into the Registrational Trial De-risk Embolden Outcomes Responders in Phase 1/2 Trial Exceed the Bar for Success Established as the Primary Endpoint in Key Design Elements Carried Into Embolden the Embolden Trial 80% Phase 1/2 Embolden Res
Slide 30: Commercial Readiness & Key Anticipated Milestones
COMMERCIAL READINESS & KEY ANTICIPATED MILESTONES
Slide 31: Building a Strong Foundation for Commercialization
Building a Strong Foundation for Commercialization Positioning Embolden clinical trial Internal CMC capabilities and Payor research confirms sites for rapid conversion manufacturing facility to strong reimbursement to commercial sites at launch produce commercial product potential for NGN-401 Separa
Slide 32: Wholly Owned and Fully Integrated In-House AAV Manufacturing
Wholly Owned and Fully Integrated In-House AAV Manufacturing GMP 42,000 sq ft facility in Houston, with 6,000 sq ft of cleanrooms Manufacturing Analytical Toxicology Batch Development Manufacturing Quality Process Control Development Flexibility to manufacture AAV product at low cost Own product qua
Slide 33: NGN-401 is Poised to Transform Treatment for Rett Syndrome
NGN-401 is Poised to Transform Treatment for Rett Syndrome TODAY UPCOMING ANTICIPATED MILESTONES Compelling Clinical Data Completion of PPQ runs by year-end 2026 Strong Cash Showed NGN-401 Balance into 1Q 29 Restarts the Development Trajectory Expected to fund Topline Data from Embolden operations t
Slide 34: Appendix: Additional NGN-401 Data
APPENDIX: ADDITIONAL NGN-401 DATA
Slide 35: RSGMS Phase 1/2 Data Showed Meaningful Gains in Gross Motor Function
RSGMS Phase 1/2 Data Showed Meaningful Gains in Gross Motor Function Mean RSGMS Total-Score Gain Exceeded Natural History Overview of Rett Syndrome Gross Motor Scale (RSGMS) Normalized to One Year Rate Validated clinical tool that assesses 15 key gross motor abilities Post Treatment (N=10) 5.2-point
Slide 36: RSHFS Phase 1/2 Data Showed Meaningful Gains in Hand Function
RSHFS Phase 1/2 Data Showed Meaningful Gains in Hand Function Overview of Rett Syndrome Hand Function Scale (RSHFS) Mean RSHFS Total-Score Gains Exceed Natural History Validated clinical tool that assesses levels of hand function 100% improved Post Treatment (N=10; 1-2.5 years) specific to Rett synd
Slide 37: ICV Delivery Achieves Greater Expression in the Brain Compared to IT-L
ICV Delivery Achieves Greater Expression in the Brain Compared to IT-L ICV route of delivery has been shown in preclinical models to have greater targeting of brain and nervous system regions underlying Rett syndrome pathophysiology compared to IT-L An estimated 30,000 ICV procedures are performed b