General Information of Drug (ID: DM6ZXAX)

Drug Name
AVR-RD-02 Drug Info
Indication
Disease Entry ICD 11 Status REF
Gaucher disease 5C56.0Y Phase 2/3 [1]
Cross-matching ID
TTD Drug ID
DM6ZXAX

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Clinical Trial Drug(s)
Drug Name Drug ID Indication ICD 11 Highest Status REF
PR001 DMQWR4N Gaucher disease 5C56.0Y Phase 1/2 [2]

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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

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.
2 Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations. J Parkinsons Dis. 2021;11(s2):S183-S188.