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:
Dec. 10, 2024
Filed:
Dec. 05, 2018
Applicant:
Vertex Pharmaceuticals Incorporated, Boston, MA (US);
Inventors:
Ewelina Morawa, Cambridge, MA (US);
Tirtha Chakraborty, Cambridge, MA (US);
Ante Sven Lundberg, Cambridge, MA (US);
Tony Ho, Cambridge, MA (US);
Laura Sandler, Cambridge, MA (US);
Brenda Eustace, Boston, MA (US);
Jerome Rossert, Boston, MA (US);
Robert Kauffman, Boston, MA (US);
Assignee:
Vertex Pharmaceuticals Incorporated, Boston, MA (US);
Attorney:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
A61K 35/28 (2015.01); A61K 31/255 (2006.01); A61K 31/395 (2006.01); A61K 35/18 (2015.01); A61K 38/19 (2006.01); A61P 7/00 (2006.01); C12N 5/0789 (2010.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01);
U.S. Cl.
CPC ...
A61K 35/28 (2013.01); A61K 31/255 (2013.01); A61K 31/395 (2013.01); A61K 35/18 (2013.01); A61K 38/193 (2013.01); A61P 7/00 (2018.01); C12N 5/0647 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 2310/20 (2017.05); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01);
Abstract
Provided herein, in some embodiments, are methods and compositions for treatment of subjects with β-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.