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US 11,453,878 B2Granted
RNA-editing oligonucleotides and uses thereof
AssigneeKorro Bio, Inc.
InventorsAndrew W. Fraley, Steven Robinette, Nessan Bermingham, Mallikarjuna Reddy Putta
Life of this patent
Estimating the term…
Abstract
The present disclosure features useful compositions and methods to treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.
Claims18
A method for deamination of an adenosine in an mRNA, the method comprising contacting a cell with an oligonucleotide comprising the structure:
- [Am]-X1-X2-X3—[Bn]
- wherein each of A and B is a nucleotide;
- m and n are each, independently, an integer from 1 to 50, and A and B combined consist of 18 to 80 nucleotides;
- X1, X2, and X3 are each, independently, a nucleotide, wherein at least one of X1, X2, and X3 has the structure of any one of Formula I-IV:
- wherein N1 is hydrogen or a nucleobase;
- R6 is hydrogen, hydroxy, or halogen;
- R7 is hydrogen, hydroxy, halogen, or C1-C6 alkoxy;
- R8 is hydrogen or halogen;
- R9 is hydrogen or hydroxy, halogen, or C1-C6 alkoxy;
- R10 is hydrogen or halogen;
- and
- R11 is hydrogen or hydroxy, halogen, or C1-C6 alkoxy,
- wherein when X1 has the structure of any one of Formulas I to IV, each of X2 and X3 is a ribonucleotide;
- when X2 has the structure of any one of Formulas I to IV, each of X1 and X3 is a ribonucleotide;
- when X3 has the structure of any one of Formulas I to IV, each of X1 and X2 is a ribonucleotide;
- when X1 and X2 each have the structure of any one of Formulas I to IV, X3 is a ribonucleotide;
- when X1 and X3 each have the structure of any one of Formulas I to IV, X2 is a ribonucleotide;
- and when X1 and X3 each have the structure of any one of Formulas I to IV, X1 is a ribonucleotide.
15 more elements
A method of treating a disorder in a subject in need thereof, the method comprising administering to the subject an effective amount of an oligonucleotide comprising the structure:
- [Am]-X1-X2-X3—[Bn]
- wherein each of A and B is a nucleotide;
- m and n are each, independently, an integer from 1 to 50, and A and B combined consist of 18 to 80 nucleotides;
- X1, X2, and X3 are each, independently, a nucleotide, wherein at least one of X1, X2, and X3 has the structure of any one of Formula I-IV:
- wherein N1 is hydrogen or a nucleobase;
- R6 is hydrogen, hydroxy, or halogen;
- R7 is hydrogen, hydroxy, halogen, or C1-C6 alkoxy;
- R8 is hydrogen or halogen;
- R9 is hydrogen or hydroxy, halogen, or C1-C6 alkoxy;
- R10 Is hydrogen or halogen;
- and
- R11 is hydrogen or hydroxy, halogen, or C1-C6 alkoxy,
- wherein when X1 has the structure of any one of Formulas I to IV, each of X2 and X3 is a ribonucleotide;
- when X2 has the structure of any one of Formulas I to IV, each of X1 and X3 is a ribonucleotide;
- when X3 has the structure of any one of Formulas I to IV, each of X1 and X2 is a ribonucleotide;
- when X1 and X2 each have the structure of any one of Formulas I to IV, X3 is a ribonucleotide;
- when X1 and X3 each have the structure of any one of Formulas I to IV, X2 is a ribonucleotide;
- and when X2 and X3 each have the structure of any one of Formulas I to IV, X1 is a ribonucleotide.
15 more elements
Dependencies are inferred from the claim text.