Last update 21 Nov 2024

Duvalgagene otiparvovec

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Target
Mechanism
GLA stimulants(α-Galactosidase stimulants)
Active Indication
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationFast Track (US)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Fabry DiseasePhase 2
US
01 Sep 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 1/2
5
(nzuozkpyom) = 4D-310 demonstrated clinically meaningful improvements on multiple cardiac endpoints through 12-24 months in five evaluable patients Cardiac biopsies from one patient at week 6 and 24 showed robust and durable delivery, transgene expression and clearance of Gb3 substrate in cardiomyocytes mkzrpvnhwc (hzwpurupkg )
Positive
29 Feb 2024
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Approval

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Regulation

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