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.

Date of Patent:
Oct. 07, 2025

Filed:

Jul. 15, 2024
Applicant:

Seattle Children's Hospital, Seattle, WA (US);

Inventors:

Carol Hsing Miao, Seattle, WA (US);

David J. Rawlings, Seattle, WA (US);

Chong Li, Seattle, WA (US);

Assignee:

Seattle Children's Hospital, Seattle, WA (US);

Attorneys:
Primary Examiner:
Assistant Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); A61K 31/573 (2006.01); A61K 39/395 (2006.01); A61P 7/04 (2006.01); C07K 16/28 (2006.01);
U.S. Cl.
CPC ...
A61K 48/0058 (2013.01); A61K 31/573 (2013.01); A61K 39/3955 (2013.01); A61K 48/0075 (2013.01); A61P 7/04 (2018.01); C07K 16/2815 (2013.01);
Abstract

Methods, compositions, and systems for treating subject(s) in need of plasma Factor VIII, particularly a subject having preexisting anti-FVIII inhibitory antibodies, are provided. The methods involve administering to the subject a therapeutically effective amount of an inflammation suppressor, a therapeutically effective amount of a CD8+ T cell depleting agent, and a therapeutically effective amount of a composition comprising a lentiviral vector (LV) comprising an optimized FVIII expression cassette expressibly linked to a megakaryocyte-specific promoter. Such methods, compositions, and systems are useful to treat subjects with blood clotting disorder(s), such as hemophilia A.


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