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US 12,115,230 B2Granted
Base editing of ANGPTL3 and methods of using same for treatment of disease
AssigneeVerve Therapeutics, Inc.
InventorsAlexandra Chadwick, Kallanthottathil G. Rajeev, Ellen Rohde, Christopher Cheng, Caroline Reiss
Life of this patent
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Abstract
Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.
Claims22
A composition for editing a gene target comprising:
- (i) an mRNA encoding a base editor protein comprising a DNA binding domain and a deaminase, wherein the mRNA comprises a sequence having at least 95% sequence identity to SEQ ID NO:
- 2192, and
- (ii) a guide RNA comprising a tracr sequence that serves as a binding scaffold for the base editor protein, and a spacer sequence that corresponds to a protospacer on ANGPTL3.
A composition for editing a gene target comprising:
- (i) an mRNA encoding a base editor protein comprising a DNA binding domain and a deaminase, wherein the mRNA comprises a sequence having at least 95% sequence identity to the coding sequence of SEQ ID NO:
- 2192, and
- (ii) a guide RNA comprising a tracr sequence that serves as a binding scaffold for the base editor protein, and a spacer sequence that corresponds to a protospacer ANGPTL3.
Dependencies are inferred from the claim text.