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
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.