Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMSCGM8)

• Drug Name: LentiGlobin Drug Info
• Synonyms: Globin gene therapy (LentiPak, sickle cell/beta-thalassemia), Genetix; Globin gene therapy (LentiPak, sickle cell/beta-thalassemia), bluebird

Indication

Disease Entry	ICD 11	Status	REF
Beta thalassemia	3A50.2	Phase 3	[1]
Sickle-cell disorder	3A51	Phase 1	[2]

• Cross-matching ID: TTD Drug ID: DMSCGM8

Molecule-Related Drug Atlas

Drug(s) Targeting Hemoglobin subunit beta (HBB)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
OTL-300	DMYGK1D	Beta thalassemia	3A50.2	Phase 1/2	[4]
PF-07059013	DMSDVKK	Sickle-cell disorder	3A51	Phase 1	[5]

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Hemoglobin subunit beta (HBB)	TTM6HK1	HBB_HUMAN	Modulator	[3]

References

• [1] ClinicalTrials.gov (NCT03207009) A Study Evaluating the Efficacy and Safety of the LentiGlobin BB305 Drug Product in Subjects With Transfusion-Dependent beta-Thalassemia. U.S. National Institutes of Health.
• [2] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [3] Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of beta-thalassemia and sickle cell disease. Curr Gene Ther. 2015;15(1):64-81.
• [4] Clinical pipeline report, company report or official report of Orchard Therapeutics.
• [5] PF-07059013: A non-covalent hemoglobin modulator favorably impacts disease state in a mouse model of sickle cell disease. Am J Hematol. 2021 Aug 1;96(8):E272-E275.