Last update 21 Nov 2024

NGGT-002

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Target
Mechanism
PAH modulators(Phenylalanine hydroxylase modulators)
Active Indication
Inactive Indication-
Inactive Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationOrphan Drug (US)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
PhenylketonuriasPhase 2
CN
11 Jul 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Early Phase 1
Phenylketonurias
PAH mutations
4
(pusbcuffkh) = The most common treatment-related adverse events (AEs) reported in all subjects were transient and asymptomatic elevations in liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST), which were well-managed by a tapering course of corticosteroid treatment. No severe liver dysfunction or long-term AEs on liver function was observed. iboxfuapxl (wymnwqdikn )
Positive
07 May 2024
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Regulation

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