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US 2025/0084408 A1Published application
Engineered rnas
AssigneeShape Therapeutics Inc.
InventorsSusan BYRNE, Brian John BOOTH, Richard Thomas SULLIVAN, Adrian Wrangham BRIGGS, Yiannis SAVVA
Life of this patent
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Abstract
Disclosed herein are engineered RNAs and compositions comprising the same for treatment of diseases or conditions in a subject. Also disclosed herein are methods of treating diseases or conditions in a subject by administering engineered RNAs, polynucleotides encoding the described engineered RNAs, or pharmaceutical compositions described here.
Claims27
-110. (canceled)
An engineered RNA, comprising:
- (a) a targeting sequence with complementarity to a target RNA;
- (b) an RNA element that comprises:
- (i) an engineered SmOPT variant sequence having at least 80% identity to SEQ ID NO:
- 87, SEQ ID NO:
- 84, SEQ ID NO:
- 85, or SEQ ID NO:
- 86;
- and
- (ii) an engineered U7 hairpin variant sequence having SEQ at least 80% identity to SEQ ID NO:
- 96, SEQ ID NO:
- 90, SEQ ID NO:
- 91, SEQ ID NO:
- 92, SEQ ID NO:
- 93, SEQ ID NO:
- 94, SEQ ID NO:
- 95, SEQ ID NO:
- 97, SEQ ID NO:
- 98, or SEQ ID NO:
- 99.
16 more elements
A polynucleotide encoding an engineered RNA wherein the engineered RNA comprises:
- (a) a targeting sequence with complementarity to a target RNA;
- (b) an RNA element that comprises:
- (iii) an engineered SmOPT variant sequence having at least 80% identity to SEQ ID NO:
- 87, SEQ ID NO:
- 84, SEQ ID NO:
- 85, or SEQ ID NO:
- 86;
- and
- (iv) an engineered U7 hairpin variant sequence having SEQ at least 80% identity to SEQ ID NO:
- 96, SEQ ID NO:
- 90, SEQ ID NO:
- 91, SEQ ID NO:
- 92, SEQ ID NO:
- 93, SEQ ID NO:
- 94, SEQ ID NO:
- 95, SEQ ID NO:
- 97, SEQ ID NO:
- 98, or SEQ ID NO:
- 99.
16 more elements
Dependencies are inferred from the claim text.