Invention Grant
- Patent Title: Allele specific modulators of P23H rhodopsin
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Application No.: US17327469Application Date: 2021-05-21
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Publication No.: US11744846B2Publication Date: 2023-09-05
- Inventor: Susan F. Murray , Punit P. Seth , Michael L. McCaleb , Susan M. Freier , Priyam Singh
- Applicant: Ionis Pharmaceuticals, Inc.
- Applicant Address: US CA Carlsbad
- Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee: Ionis Pharmaceuticals, Inc.
- Current Assignee Address: US CA Carlsbad
- Agency: Knobbe, Martens, Olson & Bear, LLP
- Main IPC: A61K31/713
- IPC: A61K31/713 ; A61P27/02 ; C07K14/705 ; A61K31/475 ; A61K31/704

Abstract:
The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.
Public/Granted literature
- US20210346421A1 ALLELE SPECIFIC MODULATORS OF P23H RHODOPSIN Public/Granted day:2021-11-11
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