Details of the Drug

General Information of Drug (ID: DMR4CX3)

Drug Name

RP-L401

Indication

Disease Entry	ICD 11	Status	REF
Infantile malignant osteopetrosis	LD24.10	Phase 1	[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
T-cell immune regulator 1 (TCIRG1)	TTVRN05	VPP3_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 (NCT04525352) A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene. U.S.National Institutes of Health.