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:
Nov. 09, 2021

Filed:

Dec. 20, 2018
Applicant:

Translate Bio, Inc., Lexington, MA (US);

Inventors:

Kim Askew, Lexington, MA (US);

Jou-Ku Chung, Lexington, MA (US);

Frank DeRosa, Lexington, MA (US);

Michael Heartlein, Lexington, MA (US);

Shrirang Karve, Lexington, MA (US);

Thomas McCauley, Lexington, MA (US);

Lianne Smith, Lexington, MA (US);

Ann J. Barbier, Lexington, MA (US);

Assignee:

TRANSLATE BIO, INC., Lexington, MA (US);

Attorneys:
Primary Examiner:
Int. Cl.
CPC ...
A61K 48/00 (2006.01); C12N 15/85 (2006.01); C12N 15/52 (2006.01); A61K 47/69 (2017.01); A61K 9/127 (2006.01); A61K 9/00 (2006.01); A61K 38/45 (2006.01); A61P 3/00 (2006.01);
U.S. Cl.
CPC ...
A61K 48/005 (2013.01); A61K 9/0019 (2013.01); A61K 9/1272 (2013.01); A61K 38/45 (2013.01); A61K 47/6929 (2017.08); A61K 48/0025 (2013.01); A61K 48/0075 (2013.01); A61K 48/0083 (2013.01); A61P 3/00 (2018.01); C12N 15/52 (2013.01); C12N 15/85 (2013.01); C12Y 201/03003 (2013.01);
Abstract

The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.


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