ABCA4 trans-splicing molecules
US12410440B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Nov 22, 2023 |
| Grant date | Sep 9, 2025 |
| Priority date | — |
| Expiry date | Nov 22, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2830/42
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.