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. 24, 2019
Filed:
Sep. 23, 2016
Applicant:
Biomarin Pharmaceutical Inc., Novato, CA (US);
Inventors:
Stuart Bunting, Novato, CA (US);
Peter Cameron Colosi, Novato, CA (US);
Erno Pungor, Novato, CA (US);
Assignee:
BIOMARIN PHARMACEUTICAL INC., Novato, CA (US);
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
A61K 38/37 (2006.01); A61K 48/00 (2006.01); C07K 14/755 (2006.01); C12N 7/00 (2006.01); C12N 15/867 (2006.01); C12N 15/00 (2006.01); A61K 47/02 (2006.01); A61K 47/10 (2017.01); A61K 47/26 (2006.01); G01N 33/68 (2006.01); A61K 39/12 (2006.01);
U.S. Cl.
CPC ...
A61K 38/37 (2013.01); A61K 39/12 (2013.01); A61K 47/02 (2013.01); A61K 47/10 (2013.01); A61K 47/26 (2013.01); A61K 48/0008 (2013.01); A61K 48/0075 (2013.01); C07K 14/755 (2013.01); C12N 7/00 (2013.01); G01N 33/6854 (2013.01); C12N 2710/16044 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14141 (2013.01); C12N 2750/14143 (2013.01); G01N 2333/015 (2013.01);
Abstract
The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.