Last update 25 Jan 2025

AMT-110

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AMT 110, AMT110, RAAV2/5-HNAGLU
Target
NAGLU
Mechanism
NAGLU replacements(Alpha-N-acetylglucosaminidase replacements), Gene transference
Therapeutic Areas
Other DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Mucopolysaccharidosis III
Inactive Indication
Lower Urinary Tract Symptoms
Originator Organization
Institut PasteurInstitut PasteurAmsterdam Molecular Therapeutics BV
Active Organization
Institut PasteurInstitut Pasteur
Inactive Organization
Recordati SpARecordati SpAuniQure biopharma BV
Drug Highest PhaseDiscovery
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with AMT-110
NCT03300453
/ CompletedPhase 1/2
Protocol AMT110-CD-001: A Phase I/II, Open-label, Study of Intracerebral Administration of Adeno-associated Viral Vector Containing the Human Alpha-N-acetylglucosaminidase cDNA in Children With Sanfilippo Type B Syndrome
100 Clinical Results associated with AMT-110
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100 Translational Medicine associated with AMT-110
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100 Patents (Medical) associated with AMT-110
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100 Deals associated with AMT-110
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Lower Urinary Tract SymptomsPhase 3
DE
Recordati SpARecordati SpA
01 Sep 2013
Lower Urinary Tract SymptomsPhase 3
IT
Recordati SpARecordati SpA
01 Sep 2013
Lower Urinary Tract SymptomsPhase 3
PL
Recordati SpARecordati SpA
01 Sep 2013
Lower Urinary Tract SymptomsPhase 3
ES
Recordati SpARecordati SpA
01 Sep 2013
Lower Urinary Tract SymptomsPhase 3
GB
Recordati SpARecordati SpA
01 Sep 2013
Mucopolysaccharidosis IIIPhase 2
FR
uniQure biopharma BV
17 Sep 2013
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Clinical Result

Indication
Phase
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Approval

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Regulation

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