Details of the Drug

General Information of Drug (ID: DM6ZXAX)

Drug Name

AVR-RD-02

Indication

Disease Entry	ICD 11	Status	REF
Gaucher disease	5C56.0Y	Phase 2/3	[1]
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Drug Type

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Lysosomal acid glucosylceramidase (GBA1)	TTCYHJ4	GBA1_HUMAN	Not Available	[1]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT05815004) Guard3: An Open-label, Parallel-arm, Randomized, Controlled, Phase 2/Phase 3 Study Evaluating the Efficacy and Safety of Autologous HSC Gene Therapy, AVR-RD-02, Compared to ERT for Gaucher Disease Type 3 in Participants Aged 2 to 25. U.S.National Institutes of Health.