Details of the Drug

General Information of Drug (ID: DMDEDV3)

Drug Name
RP-L301
Indication
Disease Entry ICD 11 Status REF
Pyruvate kinase deficiency 5C53.00 Phase 1 [1]
Drug Type
Gene therapy
Cross-matching ID
DrugBank ID
DB17661
TTD ID
D6DO9A

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Pyruvate kinase PKLR (PKLR) TT31N4S KPYR_HUMAN Not Available [1]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT04105166) Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD. U.S.National Institutes of Health.