Invention Grant
- Patent Title: Trans-splicing molecules
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Application No.: US17047496Application Date: 2019-04-17
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Publication No.: US11993776B2Publication Date: 2024-05-28
- Inventor: Philip R. Johnson , Bruce C. Schnepp , Jean Bennett , Scott J. Dooley , Krishna Jawaharlal Fisher , Junwei Sun
- Applicant: Ascidian Therapeutics, Inc. , The Trustees of the University of Pennsylvania
- Applicant Address: US MA Boston
- Assignee: Ascidian Therapeutics, Inc.,The Trustees of the University of Pennsylvania
- Current Assignee: Ascidian Therapeutics, Inc.,The Trustees of the University of Pennsylvania
- Current Assignee Address: US MA Boston; US PA Philadelphia
- Agency: Clark & Elbing LLP
- International Application: PCT/US2019/027981 2019.04.17
- International Announcement: WO2019/204514A 2019.10.24
- Date entered country: 2020-10-14
- Main IPC: C12N15/113
- IPC: C12N15/113 ; C07K14/705 ; C12Q1/68

Abstract:
The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.
Public/Granted literature
- US20210155938A1 TRANS-SPLICING MOLECULES Public/Granted day:2021-05-27
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