Uniqure N.v. · NASDAQ:QURE
Uniqure — Investor Presentation (January 2026)
34 slides from Uniqure's investor presentation, Jan 13, 2026
Slide 1: Leadership in Gene Therapy January 2026
Sophia – Huntington’s Disease Community Advocate Leadership in Gene Therapy January 2026 Exhibit 99.1
Slide 2: Disclaimer
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 2 Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other th
Slide 3: Our mission is to reimagine the future of medicine
Our mission is to reimagine the future of medicine by delivering innovative cures that transform lives. Lena - Temporal Lobe Epilepsy (TLE) Community Advocate
Slide 4: uniQure – A Leader in Gene Therapy
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 4 uniQure – A Leader in Gene Therapy *Cash, cash equivalents and investment securities. Gene therapy pioneer with validated AAV platform and successful track record AMT-130 is the first potential disease-modifying therapy for HD with blockbuster potential Le
Slide 5: Management Team
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 5 Management Team Matt Kapusta Chief Executive Officer Walid Abi-Saab, M.D. Chief Medical Officer Amin Abujoub, Ph.D. Chief Technology Operations Officer Erin Boyer Chief People and Culture Officer Christian Klemt Chief Financial Officer Richard Porter, Ph.D
Slide 6: History of Innovation
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 6 History of Innovation uniQure: A gene therapy pioneer with a 25-year history and deeply ingrained culture of innovation across an increasingly validated platform First First First First First First approved gene therapy in the western world First Commercia
Slide 7: Our Research and Development Pipeline
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 7 Our Research and Development Pipeline Preclinical Phase 1/2 Phase 3 APPROVED INDICATIONS HEMOPHILIA B etranacogene dezaparvovec (AMT-061) HUNTINGTON’S DISEASE (AMT-130) MESIAL TEMPORAL LOBE EPILEPSY (AMT-260) FABRY DISEASE (AMT-191) AMYOTROPHIC LATERAL SCL
Slide 8: AMT-130: Huntington's disease
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 8 Charles – Huntington’s Disease Community Advocate AMT-130: Huntington’s disease
Slide 9: AMT-130: Huntington's disease
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 9 • HD is a progressive neurodegenerative disease with no disease-modifying treatments available • Autosomal dominant inherited disorder (50% risk if a parent has HD) • Estimated ~100K1 genetically identifiable patients in US with HD AMT-130: Huntington’s di
Slide 10: Slowing Progression of Huntington's Disease Could Extend Patients' Quality of Life
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 10 Slowing Progression of Huntington’s Disease Could Extend Patients’ Quality of Life Untreated disease course HD is a progressive neurodegenerative disease with no disease-modifying treatments available. To slow the rate of disease progression To provide HD
Slide 11: AMT-130: A Promising Approach to Treat Huntington's Disease
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 11 AMT-130: A Promising Approach to Treat Huntington’s Disease Abbreviations: HD, Huntington’s Disease; HTT, Huntingtin protein. References. Data on file. The construct design and targeted administration of AMT-130 provide key advantages • One-time administr
Slide 12: AMT-130 Phase I/II 36-Month Data
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 12 AMT-130 Phase I/II 36-Month Data
Slide 13: AMT-130: Pivotal Phase I/II Study Design
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 13 AMT-130: Pivotal Phase I/II Study Design Prespecified statistical analysis plan High Dose AMT-130 Arm (N=17) ENROLL-HD Matched External Control Arm (N=940) 12 Months 24 Months 36 Months N=12 patients with 36-months of follow-up as of June 30, 2025 Propens
Slide 14: High-dose AMT-130 met primary and key secondary endpoints at 36 months
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 14 Generally well-tolerated with no new SAEs related to AMT-130 Safety Profile Neurodegeneration CSF NfL below baseline Statistically significant slowing of disease progression as measured by cUHDRS (primary endpoint) and TFC (secondary endpoint) Clinical Me
Slide 15: Significant slowing of disease progression by 75% based on cUHDRS
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 15 AMT-130 high-dose significantly reduced disease progression by 75% based on cUHDRS compared to a propensity score-matched external control. (p=0.003) Data cutoff date of June 30, 2025 Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Sc
Slide 16: Significant slowing of disease progression by 60% based on TFC
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 16 AMT-130 high-dose significantly reduced disease progression by 60% based on TFC compared to a propensity score-matched external control. (p=0.033) Data cutoff date of June 30, 2025 Abbreviations: cUHDRS, composite Unified Huntington’s Disease Rating Scale
Slide 17: Demonstrated reductions of CSF NfL at 36 months
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 17 AMT-130 low and high dose CSF NfL at 36 months were below baseline. *1 of 12 patients declined to undergo a lumbar puncture procedure Data cutoff date of June 30, 2025 Abbreviations: CSF, cerebrospinal fluid; NfL, neurofilament light chain References: Dat
Slide 18: Safety Summary: AMT-130 Remained Generally Well Tolerated
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 18 Safety Summary: AMT-130 Remained Generally Well Tolerated AMT-130 remained generally well tolerated, with a manageable safety profile at both doses The majority of drug-related serious adverse events occurred within the first weeks post treatment and full
Slide 19: AMT-260: Refractory Mesial Temporal Lobe Epilepsy (MTLE)
AMT-260: Refractory Mesial Temporal Lobe Epilepsy (MTLE)
Slide 20: AMT-260: Refractory Mesial Temporal Lobe Epilepsy (MTLE)
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 20 • Most common form of epilepsy • ~240K1 are treatment-resistant • AAV9-GRiK2 (miRNA) investigational gene therapy AMT-260: Refractory Mesial Temporal Lobe Epilepsy (MTLE) References: 1 Yang L et al. Long-term prognosis in temporal lobe epilepsy. Ann Palli
Slide 21: AMT-260 Phase 1/2 Overview
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 21 • Full enrollment of Cohort 1, First patient enrolled in Cohort 2 • Objective: assess safety, tolerability and signs of efficacy • Part I: U.S., multicenter, open-label, dose-finding study in a total of 12 patients • Part II: Randomized, controlled trial
Slide 22: Case Study from the First Participant Dosed with AMT-260
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 22 Safety data: • No SAEs • No AE of neuroinflammation or neuroimaging abnormalities • No worsening seizures or new seizure type % Change in 30 day Seizure Frequency from Screening Baseline As of 4/17/25 (5 mo data preliminary) Exploratory Efficacy Data: • E
Slide 23: AMT-191: Fabry Disease
as AMT-191: Fabry Disease
Slide 24: AMT-191: Fabry Disease
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 24 • ~15,0001 people in US+EU5 • ERT – poor uptake in heart/kidneys • AAV5-GLA investigational gene therapy AMT-191: Fabry Disease Reference: 1. Spada, et al, Am. J. Hum. Gent. 2006:79, 31-40 Abbreviations: EU5, Germany, France, Italy, Spain United Kingdom;
Slide 25: AMT-191 Phase 1/2 Overview
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 25 Dose Cohort 1 Patient 1 DSMB Patient 2 Patient 3 Patient 4 - 6 DSMB Dose Cohort 2 Patient 1 DSMB Patient 2 Patient 3 Patient 4 - 6 DSMB Dose Cohort 3 Patient 1 DSMB Patient 2 Patient 3 Patient 4 - 6 DSMB • Cohorts 1, 2 and 3 are fully enrolled • Objective
Slide 26: Plasma α-GAL Activity
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 26 • Achievement of supraphysiological* α-GAL activity observed after 1- week post-treatment • Sustained elevated α-GAL levels for up to 45 weeks • Therapeutic levels maintained post-ERT discontinuation * Normal α-GAL range: 1.38-8.66 nmol, Mean normal 3.57
Slide 27: AMT-191 Cohort 1 Exploratory Efficacy Biomarkers
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 27 • Lyso-Gb3 levels were higher than normal range* however in line with patients receiving ERT • Lyso-Gb3 levels remained stable post AMT-191 administration * Normal lyso-Gb3 range: < 2.7 ng/mL ** Data cutoff date of July 24, 2025 Abbreviations: ERT, enzyme
Slide 28: AMT-191 Safety Data
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 28 AMT-191: Cohort 1 – Safety Data Manageable Safety Profile at 6.0x1013 gc/kg Dose Treatment-emergent adverse events: Majority Grade 1-2 laboratory values. A single Grade 3 laboratory value (LFT elevation) – resolved with corticosteroids Of TEAEs reported,
Slide 29: Key Milestones
Key Milestones Ashley – Huntington’s Disease Community Advocate
Slide 30: Key Milestones Achieved in 2025
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 30 Key Milestones Achieved in 2025 AMT-130: Huntington’s Disease Presented case study from first patient dosed with AMT-260 MTLE in 2Q25 Presented initial data from AMT-191 Fabry study in 3Q25 Abbreviations: BLA, Biologics License Application; FDA, Food &
Slide 31: Planned Milestones for 2026
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 31 Planned Milestones for 2026 Abbreviations: BLA, Biologics License Application; FDA, Food & Drug Administration; MTLE, mesial temporal lobe epilepsy; CMC, Chemistry, Manufacturing and Controls Other Programs • Present additional clinical data in AMT-260 MT
Slide 32: Strong Financial Position with Prudent Capital Allocation
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 32 Strong Financial Position with Prudent Capital Allocation *Cash, cash equivalents and investment securities In September 2025, completed an upsized $300 million public offering with gross proceeds of $345 million. Also in September 2025, refinanced existi
Slide 33: uniQure – A Leader in Gene Therapy
LEADERSHIP IN GENE THERAPY JANUARY 2026 | 33 uniQure – A Leader in Gene Therapy *Cash, cash equivalents and investment securities. Gene therapy pioneer with validated AAV platform and successful track record AMT-130 is the first potential disease-modifying therapy for HD with blockbuster potential L
Slide 34: uniQure
uniQure