Invention Grant
- Patent Title: Pharmaceutical composition and treatment method for genetic disease associated with splicing abnormalities
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Application No.: US17210874Application Date: 2021-03-24
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Publication No.: US12023338B2Publication Date: 2024-07-02
- Inventor: Masatoshi Hagiwara , Masahiko Ajiro
- Applicant: KYOTO UNIVERSITY
- Applicant Address: JP Kyoto
- Assignee: KYOTO UNIVERSITY
- Current Assignee: KYOTO UNIVERSITY
- Current Assignee Address: JP Kyoto
- Agency: Birch, Stewart, Kolach & Birch, LLP
- Priority: JP 17029306 2017.02.20
- The original application number of the division: US16486980
- Main IPC: A61K31/519
- IPC: A61K31/519 ; A61K31/428 ; A61K31/437 ; A61K31/443 ; A61K31/4439 ; A61K31/4709 ; A61K31/52 ; A61K31/5377 ; A61K31/55 ; A61P11/00 ; A61P43/00
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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