Last update 07 Mar 2025

uniQure biopharma BV

Subsidiary Company|
Netherlands

Overview

Tags

Nervous System Diseases
Other Diseases
Congenital Disorders
AAV based gene therapy

Disease domain score
A glimpse into the focused therapeutic areas
Technology Platform
Most used technologies in drug development
Targets
Most frequently developed targets
Disease DomainCount
Nervous System Diseases5
Endocrinology and Metabolic Disease2
Infectious Diseases1
Top 5 Drug TypeCount
AAV based gene therapy6
Top 5 TargetCount
GPR180(G protein-coupled receptor 180)1
SOD1(Superoxide dismutase)1
α-synuclein(Synuclein alpha)1
GALA(α-Galactosidase)1
HTT(Huntingtin)1

Related

6
Drugs associated with uniQure biopharma BV
AMT-162
Target
SOD1
Mechanism
SOD1 gene inhibitors
Active Org.
uniQure biopharma BV
Originator Org.
UMass Chan Medical SchoolUMass Chan Medical School
Active Indication
Amyotrophic Lateral Sclerosis
Inactive Indication-
Drug Highest PhasePhase 1/2
First Approval Ctry. / Loc.-
First Approval Date-
AMT-260
Target
GluK2 x Kainic acid receptor
Mechanism
GluK2 modulators [+2]
Active Org.
uniQure NVuniQure NV [+1]
Originator Org.
uniQure biopharma BV
Active Indication
Epilepsy, Temporal Lobe [+1]
Inactive Indication-
Drug Highest PhasePhase 1/2
First Approval Ctry. / Loc.-
First Approval Date-
AMT-191
Target
GALA
Mechanism
GLA stimulants
Active Org.
uniQure biopharma BV [+1]
Originator Org.
uniQure NVuniQure NV
Active Indication
Fabry Disease
Inactive Indication-
Drug Highest PhasePhase 1/2
First Approval Ctry. / Loc.-
First Approval Date-
9
Clinical Trials associated with uniQure biopharma BV
NCT06100276
/ RecruitingPhase 1/2
A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
NCT06063850
/ RecruitingPhase 1/2
A Multi-center, Phase 1/2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults with Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered Via Magnetic Resonance Imaging (MRI)-Guided Convection-enhanced Delivery (CED)
NCT06270316
/ RecruitingPhase 1/2
A Phase 1/2, Single Dose, Dose Escalation Study of Intravenous AAV5-GLA (AMT-191) in Adult Males With Classic Fabry Disease
100 Clinical Results associated with uniQure biopharma BV
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0 Patents (Medical) associated with uniQure biopharma BV
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100 Deals associated with uniQure biopharma BV
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100 Translational Medicine associated with uniQure biopharma BV
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Pipeline

Pipeline Snapshot as of 26 Apr 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3
Discovery
2
1
Preclinical
Phase 2
3
2
Other
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Current Projects

Drug(Targets)IndicationsGlobal Highest Phase
AMT-130
 ( HTT )		Huntington Disease
More
Phase 1/2
AMT-162
 ( SOD1 )		Amyotrophic Lateral Sclerosis
More
Phase 1/2
AMT-191
 ( GALA )		Fabry Disease
More
Phase 1/2
AMT-260
 ( GluK2 x Kainic acid receptor )		Epilepsy
More
Preclinical
WO2023198663
 ( α-synuclein )Patent Mining		Nervous System Diseases
More
Discovery
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