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:
Jul. 08, 2025

Filed:

Mar. 03, 2021
Applicant:

Arcturus Therapeutics, Inc., San Diego, CA (US);

Inventors:

Padmanabh Chivukula, San Diego, CA (US);

Priya Prakash Karmali, San Diego, CA (US);

Kiyoshi Tachikawa, San Diego, CA (US);

Suezanne E. Parker, Ramona, CA (US);

Marciano Rodriguez Sablad, San Diego, CA (US);

Pattraranee Limphong, San Diego, CA (US);

Yanjie Bao, San Diego, CA (US);

Jerel Boyd Lee Vega, San Diego, CA (US);

Assignee:

Arcturus Therapeutics, Inc., San Diego, CA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C07H 21/02 (2006.01); A61K 9/1272 (2025.01); A61K 31/221 (2006.01); A61K 48/00 (2006.01); C07H 21/04 (2006.01); C12N 9/10 (2006.01); A61K 9/1271 (2025.01); A61K 39/00 (2006.01);
U.S. Cl.
CPC ...
C12N 9/1018 (2013.01); A61K 9/1272 (2013.01); A61K 31/221 (2013.01); A61K 48/00 (2013.01); A61K 48/0033 (2013.01); A61K 9/1271 (2013.01); A61K 2039/51 (2013.01); C12Y 201/03003 (2013.01);
Abstract

The present disclosure describes compositions and methods for treating ornithine transcarbamylase (OTC) deficiency. The compositions include a lipid formulation and messenger RNA (mRNA) encoding an OTC enzyme. The lipid formulations can comprise an ionizable cationic lipid in a lipid nanoparticle encapsulating the mRNA.


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