Slide 1: Huntington's Disease Program Update
Ashley – Huntington’s Disease Community Advocate Phase I/II ifezuntirgene inilparvovec (AMT-130) Huntington’s Disease Program Update September 29, 2026
Slide 2: Forward-looking statements
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 2 This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact
Slide 3: Opening Remarks
Opening Remarks Matt Kapusta Chief Executive Officer
Slide 4: AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 4 AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression Substantial treatment effect at 36 months in updated analysis of all 15 high-dose patients, strengthening the data at the 36- month timepoint 48-month analysis showed co
Slide 5: Review of Data Supporting the BLA and MAA Submissions: 36 Month Analysis
Review of Data Supporting the BLA and MAA Submissions: June 2025 Data Cut: 36 Month Analysis of AMT-130 High Dose (N=12) Walid Abi-Saab, M.D. Chief Medical Officer BLA, Biologics License Application; MAA, Marketing Authorization Application
Slide 6: 36-Month Statistical Analysis Plan: Basis for BLA and MAA Submissions
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 6 36-Month Statistical Analysis Plan: Basis for BLA and MAA Submissions 12 Months 24 Months 36 Months N=12 patients with 36-months of follow-up as of June 30, 2025 Propensity score-matched to AMT-130 high-dose arm High-Dose AMT-130 Arm (N=17) ENROLL-HD A M
Slide 7: Basis for BLA and MAA Submissions
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 7 Basis for BLA and MAA Submissions Statistically significant 60% slowing based on TFC at 36 months (p=0.033) Statistically significant 75% slowing based on cUHDRS at 36 months (p=0.003) Abbreviations: BLA, Biologics License Application; MAA, Marketing Aut
Slide 8: Primary Analysis: Results at 48 Months from the Phase I/II Study
Data as of June 30, 2026 Primary Analysis: Results at 48 Months from the Phase I/II Study
Slide 9: 48-Month Statistical Analysis Plan Included Updated External Control
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 9 Enroll-HD A – Release date January 2023; Enroll-HD B – Release date September 2025 Abbreviations: HD, Huntington’s disease NCT05243017, NCT04120493 48-Month Statistical Analysis Plan Included Updated External Control High-Dose AMT-130 Arm (N=17) Updated
Slide 10: Dose Dependence Sustained Through 48 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 10 Dose Dependence Sustained Through 48 Months Above graph represents observed data. Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline References: Data on file. Sept
Slide 11: Meaningful Slowing of Disease Progression in cUHDRS and TFC at Month 48
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 11 AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression in cUHDRS and TFC at Month 48 DISEASE WORSENING Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease, SE, standard error
Slide 12: Meaningful Slowing of Disease Progression Through 48 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 12 Participants Baseline 12 months 24 months 36 months 48 months AMT-130 High-Dose 17 17 15 15 12 Enroll-HD B 1337 967 772 704 641 DISEASE WORSENING Participants Baseline 12 months 24 months 36 months 48 months AMT-130 High-Dose 17 17 15 15 12 Enroll-HD B
Slide 13: Other Secondary Endpoints at 48 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 13 Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; SDMT, Symbol Digit Modalities Test; SWRT, Stroop Word Reading Test; TMS, Total Motor Score; LSM, least squares mean; BL, baseline; SE, standard error; HD, Huntington’s disease R
Slide 14: A Closer Look at Enroll-HD B External Control Matched to High-Dose
A Closer Look at Enroll-HD B External Control Matched to High-Dose
Slide 15: Updated Matched Natural History Control Likely Understates Disease Progression
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 15 Updated Matched Natural History Control Likely Understates Disease Progression and Treatment Effect at Month 48 Enroll-HD B: Annualized cUHDRS Change from Baseline By Length of Follow Up • Participants with longer follow up declined more slowly, enrichi
Slide 16: Post Hoc Analysis using Enroll-HD A at 48 Months
Post Hoc Analysis using Enroll-HD A at 48 Months from the Phase I/II Study Data as of June 30, 2026
Slide 17: Meaningful Slowing in cUHDRS at Month 48 vs Enroll-HD A
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 17 -0.90 -1.94 -2.5 -2 -1.5 -1 -0.5 0 AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression in cUHDRS at Month 48 DISEASE WORSENING Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s
Slide 18: Meaningful Slowing of Disease Progression Through 48 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 18 DISEASE WORSENING Above graph represents observed data Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; TFC, Total Functional Capacity; SE, standard error; BL, baseline References: Data on file. September 2026 cUHDRS Change fr
Slide 19: Results at 36 Months from the Phase I/II Study
Results at 36 Months from the Phase I/II Study Data as of June 30, 2026
Slide 20: Meaningful Slowing of Disease Progression Through 36 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 20 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0.2 0.4 0.6 Baseline 12 Months 24 Months 36 Months -2.5 -2 -1.5 -1 -0.5 0 0.5 1 1.5 Baseline 12 Months 24 Months 36 Months Participants Baseline 12 months 24 months 36 months AMT-130 High-Dose 2025 17 17 15 12 AMT-130
Slide 21: Slowing Through 36 Months with Three Additional Subjects
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 21 -1.4 -1.2 -1 -0.8 -0.6 -0.4 -0.2 0 0.2 0.4 0.6 Baseline 12 Months 24 Months 36 Months -2.5 -2 -1.5 -1 -0.5 0 0.5 1 1.5 Baseline 12 Months 24 Months 36 Months Enroll-HD B Participants Baseline 12 months 24 months 36 months AMT-130 High-Dose 2026 17 17 15
Slide 22: Slowing on cUHDRS and TFC at 36 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 22 Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; HD, Huntington’s disease, TFC, Total Functional Capacity SE, standard error;; LSM, least squares mean; BL, baseline; HD, Huntington’s disease References: Data on file. September
Slide 23: Other Secondary Endpoints at 36 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 23 Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale; SDMT, Symbol Digit Modalities Test; SWRT, Stroop Word Reading Test; TMS, Total Motor Score; LSM, least squares mean; BL, baseline; SE, standard error; HD, Huntington’s disease R
Slide 24: Neurofilament Light Chain (NfL)
Neurofilament Light Chain (NfL)
Slide 25: CSF NfL Near Baseline at 48 Months
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 25 -100 0 100 200 300 400 500 0 12 24 36 48 Percentage change in CSF NfL (SE) Months CSF NfL Percentage Change from Baseline (0-48 Months) Patients Base 1M 3M 6M 9M 12M 15M 18M 24M 30M 36M 48M High-Dose 17 17 16 16 15 14 14 15 15 14 14 11 Low-Dose 12 12 11
Slide 26: uniQure
uniQure
Slide 27: AMT-130 Was Generally Well-Tolerated
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 27 AMT-130 Was Generally Well-Tolerated • Most common adverse events in the treatment groups related to the administration procedure, which all resolved • Since September 2025: - No new treatment-related serious adverse events (SAE) reported in Cohorts 1 a
Slide 28: Adverse Events Summary Table
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 28 All AMT-130 (Cohorts 1, 2, 3 and 4) (n=51) High-dose AMT-130 (Cohort 4) (n= 6) Dose-Blinded AMT-130 (Cohort 3) (n=12) High-dose AMT-130 (Cohorts 2) (n=20*) Low-dose AMT-130 (Cohorts 1) (n=13*) Sham Surgery (n=10) N (%) N (%) N (%) N (%) N (%) N (%) Any
Slide 29: Summary
Summary
Slide 30: AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression
LEADERSHIP IN GENE THERAPY SEPTEMBER 2026 | 30 AMT-130 High-Dose Continued to Demonstrate Meaningful Slowing of Disease Progression Substantial treatment effect at 36 months in updated analysis of all 15 high-dose patients, strengthening the data at the 36- month timepoint 48-month analysis showed c
Slide 31: Clinician's Perspective
Clinician’s Perspective Victor Sung, M.D. University of Alabama at Birmingham (UAB)
Slide 32: Questions
uniQure Research Ani Questions
Slide 33: Closing Remarks
Ashley – Huntington’s Disease Community Advocate Closing Remarks Matt Kapusta Chief Executive Officer
Slide 34: uniQure
uniQure