General Information of Drug (ID: DMQWR4N)

Drug Name
PR001 Drug Info
Indication
Disease Entry ICD 11 Status REF
Gaucher disease 5C56.0Y Phase 1/2 [1]
Parkinson disease 8A00.0 Phase 1/2 [2]
Cross-matching ID
TTD Drug ID
DMQWR4N

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
AVR-RD-02 DM6ZXAX Gaucher disease 5C56.0Y Phase 2/3 [4]

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 [3]

References

1 ClinicalTrials.gov (NCT04411654) An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose LY3884961 in Infants With Type 2 Gaucher Disease. U.S.National Institutes of Health.
2 ClinicalTrials.gov (NCT04127578) A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of LY3884961 in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL). U.S.National Institutes of Health.
3 Gene Therapy for Parkinson's Disease Associated with GBA1 Mutations. J Parkinsons Dis. 2021;11(s2):S183-S188.
4 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.