Last update 21 Nov 2024

AX-552

Overview

Basic Info

Drug Type
Enzyme
Synonyms
ICV-ERT(BioMarin Pharmaceutical)
Target
HS x β-galactosidase
Mechanism
Heparan sulfate sulfatase replacements, β-galactosidase replacements(Beta-galactosidase replacements)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Gangliosidosis, GM1
Inactive Indication-
Originator Organization
Allievex Corp.Allievex Corp.
Active Organization
Allievex Corp.Allievex Corp.BioMarin Pharmaceutical, Inc.BioMarin Pharmaceutical, Inc.
Inactive Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with AX-552
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100 Translational Medicine associated with AX-552
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100 Patents (Medical) associated with AX-552
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100 Deals associated with AX-552
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Gangliosidosis, GM1Preclinical
US
BioMarin Pharmaceutical, Inc.BioMarin Pharmaceutical, Inc.
01 Sep 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Approval

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Regulation

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