Last update 18 Apr 2026

AAV9-hAGA (Rare Trait Hope Fund)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
DANAGALEX, recombinant serotype 9 adeno-associated virus encoding a codon-optimized human aspartylglucosaminidase (AGA) transgene (Rare Trait Hope Fund)
Target
AGA
Action
modulators
Mechanism
AGA modulators(aspartylglucosaminidase modulators)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases
Active Indication
Aspartylglucosaminuria
Inactive Indication-
Originator Organization
Rare Trait Hope Fund
Active Organization
Rare Trait Hope Fund
Inactive Organization-
License Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationOrphan Drug (United States)
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Related

1
Clinical Trials associated with AAV9-hAGA (Rare Trait Hope Fund)
NCT07530796
/ Not yet recruitingPhase 1/2
An Open-Label, Single Center, Phase 1/2 Study to Evaluate the Safety and Efficacy of DANAGALEX (scAAV9/AGA) in Participants With Aspartylglucosaminuria (AGU)
100 Clinical Results associated with AAV9-hAGA (Rare Trait Hope Fund)
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100 Translational Medicine associated with AAV9-hAGA (Rare Trait Hope Fund)
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100 Patents (Medical) associated with AAV9-hAGA (Rare Trait Hope Fund)
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100 Deals associated with AAV9-hAGA (Rare Trait Hope Fund)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
AspartylglucosaminuriaPhase 2-
Rare Trait Hope Fund
01 May 2026
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Clinical Trial

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Approval

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Regulation

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