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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.:

US 12023338 B1

PDFFull Text
Date of Patent:
Jul. 02, 2024

Filed:

Mar. 24, 2021

Pharmaceutical composition and treatment method for genetic disease associated with splicing abnormalities

Applicant:

Kyoto University, Kyoto, JP;

Inventors:

Masatoshi Hagiwara, Kyoto, JP;

Masahiko Ajiro, Kyoto, JP;

Assignee:

KYOTO UNIVERSITY, Kyoto, JP;

Attorney:

Birch, Stewart, Kolach & Birch, LLP

Primary Examiner:

Amy L Clark

Assistant Examiner:

Janet L Coppins

Int. Cl.
CPC ...
A61K 31/519 (2006.01); A61K 31/428 (2006.01); A61K 31/437 (2006.01); A61K 31/443 (2006.01); A61K 31/4439 (2006.01); A61K 31/4709 (2006.01); A61K 31/52 (2006.01); A61K 31/5377 (2006.01); A61K 31/55 (2006.01); A61P 11/00 (2006.01); A61P 43/00 (2006.01);
U.S. Cl.
CPC ...
A61K 31/52 (2013.01); A61K 31/428 (2013.01); A61K 31/437 (2013.01); A61K 31/443 (2013.01); A61K 31/4439 (2013.01); A61K 31/4709 (2013.01); A61K 31/519 (2013.01); A61K 31/5377 (2013.01); A61K 31/55 (2013.01); A61P 11/00 (2018.01); A61P 43/00 (2018.01);
Abstract

A pharmaceutical composition for genetic diseases caused by an aberrant splicing regulation is provided. Provided are a pharmaceutical composition for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases caused by an aberrant splicing regulation, the pharmaceutical composition containing, as an active ingredient, a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation, and a method for preventing, ameliorating, suppressing progression of, and/or treating the genetic diseases using a compound capable of suppressing an aberrant splicing regulation that contributes to the development or progression of genetic diseases caused by an aberrant splicing regulation.

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