IP Library Granted Patent US 12,351,818
Granted Patent B2
US 12,351,818 · App. 18/518,831 · Granted Jul 8, 2025

Gene-modifying endonucleases

Inventors: Basem Al-Shayeb (San Francisco, CA); Jacob Borrajo (San Francisco, CA); Mohammad Kamyab Javanmardi (San Francisco, CA); Kushagra Sharma (San Francisco, CA)
Assignee: Amber Bio Inc.
C12N15/907C12N9/22C12N15/11C12N15/111C12N2310/20
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Quick Facts
Patent No.
US 12,351,818
App. No.
18/518,831
Granted
Jul 8, 2025
Kind
B2
Abstract

The disclosure relates to compositions and methods that modify target nucleic acids, as well as methods of detecting nucleic acids. Various compositions are described herein, including compositions comprising endonucleases, endonuclease systems, and chimeric proteins having the endonuclease and a nucleic-acid modulating domain or a nucleic acid modifying domain.

Claims (36)

1. A nuclease system, comprising:

(a) an endonuclease comprising a nucleic acid encoding an endonuclease comprising an endonuclease comprising a sequence, or a fragment or variant thereof, and having at least 90% identity to SEQ ID NO: 3 or having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) an RNA molecule comprising a sequence complementary to one strand of a target nucleic acid molecule,

wherein the endonuclease further comprises a nuclear localization signal (NLS).

2. The nuclease system of claim 1 , comprising a higher eukaryotes and prokaryotes nucleotide-binding domain (HEPN) domain.

3. The nuclease system of claim 1 , further comprising one or more donor polynucleotides.

4. The nuclease system of claim 1 , wherein the amino acid modifications are selected from D38F, A59V, G172L, T236L, T319I, H375L, H419Y, T424F, E529L, T541L, G562Y, K564M, D569L, A586I, N641F, D642L, S647L, D721L, R779I, K13R, K566R, G554H, A35N, E110T, G314Q, K114P, D498P, I86P, V57E, H249W, and R704F relative to SEQ ID NO: 3.

5. A chimeric protein comprising:

(a) an endonuclease comprising an endonuclease comprising a sequence, or a fragment or variant thereof, and having at least 90% identity to SEQ ID NO: 3 or having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) a nucleic acid-modulating domain or a nucleic acid-modifying domain comprising a sequence comprising a catalytic domain, or a fragment or variant thereof,

wherein (a) and (b) do not naturally occur together in a same reading frame.

6. The chimeric protein of claim 5 , wherein the nucleic acid-modulating domain or the nucleic acid-modifying domain is selected from MS binding protein (MCP), lambdaN, serine/threonine-protein phosphatase 7 (PP7), QBeta, stem-loop binding protein (SLBP), and TATA-binding protein/TAR DNA Binding Protein (TBP/TAR).

7. The chimeric protein of claim 5 , comprising a higher eukaryotes and prokaryotes nucleotide-binding domain (HEPN) domain.

8. The chimeric protein of claim 5 , further comprising one or more donor polynucleotides.

9. The chimeric protein of claim 5 , wherein the amino acid modifications are selected from D38F, A59V, G172L, T236L, T319I, H375L, H419Y, T424F, E529L, T541L, G562Y, K564M, D569L, A586I, N641F, D642L, S647L, D721L, R779I, K13R, K566R, G554H, A35N, E110T, G314Q, K114P, D498P, I86P, V57E, H249W, and R704F relative to SEQ ID NO: 3.

10. A composition comprising a complex comprising chimeric protein and an RNA molecule, wherein the chimeric protein comprises:

(a) an endonuclease comprising a sequence, or a fragment or variant thereof, and having at least 90% identity to SEQ ID NO: 3 or having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) a nucleic acid-modulating domain or a nucleic acid-modifying domain comprising a sequence comprising a catalytic domain, or a fragment or variant thereof,

wherein (a) and (b) do not naturally occur together in a same reading frame and

the RNA molecule comprises a sequence complementary to one strand of a target nucleic acid molecule.

11. The composition of claim 10 , comprising a higher eukaryotes and prokaryotes nucleotide-binding domain (HEPN) domain.

12. The composition of claim 10 , further comprising one or more donor polynucleotides.

13. The composition of claim 10 , wherein the amino acid modifications are selected from D38F, A59V, G172L, T236L, T319I, H375L, H419Y, T424F, E529L, T541L, G562Y, K564M, D569L, A586I, N641F, D642L, S647L, D721L, R779I, K13R, K566R, G554H, A35N, E110T, G314Q, K114P, D498P, 186P, V57E, H249W, and R704F relative to SEQ ID NO: 3.

14. The chimeric protein of claim 10 , wherein the nucleic acid-modulating domain or the nucleic acid-modifying domain is selected from MS binding protein (MCP), lambdaN, serine/threonine-protein phosphatase 7 (PP7), QBeta, stem-loop binding protein (SLBP), and TATA-binding protein/TAR DNA Binding Protein (TBP/TAR).

15. A nuclease system, comprising:

(a) an endonuclease comprising a nucleic acid encoding an endonuclease comprising an endonuclease comprising a sequence, or a fragment or variant thereof, and having at least 90% identity to SEQ ID NO: 3 or having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) an RNA molecule comprising a sequence complementary to one strand of a target nucleic acid molecule,

wherein the nucleic acid is operably linked to a eukaryotic or viral promoter.

16. A nuclease system, comprising:

(a) an endonuclease comprising a nucleic acid encoding an endonuclease comprising a sequence having at least 90% identity to SEQ ID NO: 3 and having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) an RNA molecule comprising a sequence complementary to one strand of a target nucleic acid molecule.

17. The nuclease system of claim 16 , wherein the amino acid modifications are selected from D38F, A59V, G172L, T236L, T319I, H375L, H419Y, T424F, E529L, T541L, G562Y, K564M, D569L, A586I, N641F, D642L, S647L, D721L, R779I, K13R, K566R, G554H, A35N, E110T, G314Q, K114P, D498P, 186P, V57E, H249W, and R704F relative to SEQ ID NO: 3.

18. A nuclease system, comprising:

(a) an endonuclease comprising a nucleic acid encoding an endonuclease comprising an endonuclease comprising a sequence, or a fragment or variant thereof, and having at least 90% identity to SEQ ID NO: 3 or having about 1 to about 20 amino acid modifications relative to SEQ ID NO: 3; and

(b) an RNA molecule comprising a sequence complementary to one strand of a target nucleic acid molecule,

wherein the target nucleic acid molecule is a eukaryotic nucleic acid molecule.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 23, 2024
From: AL-SHAYEB, BASEM; BORRAJO, JACOB; JAVANMARDI, MOHAMMAD KAMYAB; SHARMA, KUSHAGRA
To: AMBER BIO INC.
Reel/Frame 066207/0138 →
Continuity (5)
Provisional Application 63504721 · May 26, 2023
Provisional Application 63500779 · May 8, 2023
Provisional Application 63386784 · Dec 9, 2022
Provisional Application 63384937 · Nov 23, 2022
Related Publication 20240344087A1 · Oct 17, 2024
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Cited By (1)
US 12,686,869