Claims for Patent: 8,058,061

Summary for Patent: 8,058,061

Title:	Vector encoding human globin gene and use thereof in treatment of hemoglobinopathies
Abstract:	Recombinant lentiviral vectors having a region encoding a functional β-globin gene; and large portions of the β-globin locus control regions which include DNase I hypersensitive sites HS2, HS3 and HS4 provides expression of β-globin when introduced into a mammal, for example a human, in vivo. Optionally, the vector further includes a region encoding a dihydrofolate reductase. The vector may be used in treatment of hemoglobinopathies, including β-thalessemia and sickle-cell disease. For example, hematopoietic progenitor or stem cells may be transformed ex vivo and then restored to the patient. Selection processes may be used to increase the percentage of transformed cells in the returned population. For example, a selection marker which makes transformed cells more drug resistant than untransformed cells allows selection by treatment of the cells with the corresponding drug.
Inventor(s):	Michel Sadelain, Stefano Rivella, Chad May, Joseph Bertino
Assignee:	Memorial Sloan Kettering Cancer Center
Application Number:	US12/433,412

Details for Patent 8,058,061

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Bluebird Bio Inc.	ZYNTEGLO	betibeglogene autotemcel	For Injection	125717	August 17, 2022		2029-04-30

International Patent Family for US Patent 8,058,061

Country	Patent Number	Estimated Expiration
World Intellectual Property Organization (WIPO)	03002155
United States of America	7541179
United States of America	2009274671
United States of America	2003022303
Japan	2004537539