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. 14, 2023

Filed:

Apr. 21, 2023
Applicant:

Stoke Therapeutics, Inc., Bedford, MA (US);

Inventors:

Isabel Aznarez, Boston, MA (US);

Aditya Venkatesh, Natick, MA (US);

Gene Liau, Wayland, MA (US);

Assignee:

STOKE THERAPEUTICS, INC., Bedford, MA (US);

Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61P 27/02 (2006.01); A61K 9/00 (2006.01); C12N 15/86 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/0048 (2013.01); A61P 27/02 (2018.01); C12N 15/86 (2013.01); C12N 2310/11 (2013.01); C12N 2310/20 (2017.05); C12N 2310/314 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2750/14143 (2013.01);
Abstract

Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. Such therapeutic agents can be used to treat a condition or disease caused by protein deficiency and/or mitochondrial function deficit.


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