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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.:

US 12043843 B1

PDFFull Text
Date of Patent:
Jul. 23, 2024

Filed:

Nov. 04, 2016

Materials and methods for treatment of hemoglobinopathies

Applicant:

Crispr Therapeutics Ag, Basel, CH;

Inventors:

Matthew Hebden Porteus, Cambridge, MA (US);

Melanie Ruth Allen, Cambridge, MA (US);

Chad Albert Cowan, Cambridge, MA (US);

Ante Sven Lundberg, Cambridge, MA (US);

Michelle I-Ching Lin, Cambridge, MA (US);

Jeffrey Li, Cambridge, MA (US);

Thao Thi Nguyen, Cambridge, MA (US);

Assignee:

Vertex Pharmaceuticals Incorporated, Boston, MA (US);

Attorney:
Primary Examiner:

Marcia S Noble

Int. Cl.
CPC ...
C12N 15/85 (2006.01); A61P 7/00 (2006.01); C07K 14/805 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/90 (2006.01); A61K 48/00 (2006.01); C12N 15/10 (2006.01); C12N 15/113 (2010.01); C12N 15/63 (2006.01);
U.S. Cl.
CPC ...
C12N 15/85 (2013.01); A61P 7/00 (2018.01); C07K 14/805 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/907 (2013.01); A61K 48/00 (2013.01); C12N 15/102 (2013.01); C12N 15/113 (2013.01); C12N 15/63 (2013.01); C12N 2310/20 (2017.05); C12N 2800/80 (2013.01);
Abstract

The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

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