The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Jun. 02, 2026
Filed:
Oct. 26, 2018
Vertex Pharmaceuticals Incorporated, Boston, MA (US);
Tirtha Chakraborty, Cambridge, MA (US);
Bibhu Prasad Mishra, Cambridge, MA (US);
Chad Albert Cowan, Cambridge, MA (US);
Ante Sven Lundberg, Cambridge, MA (US);
Samarth Kulkarni, Cambridge, MA (US);
Todd Douglass Borland, Cambridge, MA (US);
Vertex Pharmaceuticals Incorporated, Boston, MA (US);
Abstract
Provided are materials and methods for treating patients with hemoglobinopathies, either ex vivo or in vivo. Also provided are materials and methods for deleting and/or mutating a portion of a human beta globin locus on chromosome 11 and one or more of: a BCL11 A gene on chromosome 2, and a DNA sequence that encodes a transcriptional control region of the BCL11 A gene on chromosome 2, in a human cell by genome editing and thereby increasing the production of fetal hemoglobin (HbF) in the genome-edited human cells.