Details of the Drug

General Information of Drug (ID: DMSJU5B)

Drug Name

RP-L102

Indication

Disease Entry	ICD 11	Status	REF
Fanconi's anemia	3A70.0	Phase 2	[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
Fanconi anemia group A protein (FANCA)	TTV5HJS	FANCA_HUMAN	Replacement	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT04248439) Gene Therapy for Fanconi Anemia, Complementation Group A. U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of Rocket Pharmaceuticals.