Details of the Drug

General Information of Drug (ID: DMQWR4N)

Drug Name

PR001

Indication

Disease Entry	ICD 11	Status	REF
Gaucher disease	5C56.0Y	Phase 1/2	[1]
Parkinson disease	8A00.0	Phase 1/2	[2]
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Drug Type

Gene therapy

Cross-matching ID

Repurposed Drugs (RPD)

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	[3]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

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.