US 12,351,818 B2
Gene-modifying endonucleases
Basem Al-Shayeb, San Francisco, CA (US); Jacob Borrajo, San Francisco, CA (US); Mohammad Kamyab Javanmardi, San Francisco, CA (US); and Kushagra Sharma, San Francisco, CA (US)
Assigned to Amber Bio Inc., San Francisco, CA (US)
Filed by Amber Bio Inc., San Francisco, CA (US)
Filed on Nov. 24, 2023, as Appl. No. 18/518,831.
Claims priority of provisional application 63/504,721, filed on May 26, 2023.
Claims priority of provisional application 63/500,779, filed on May 8, 2023.
Claims priority of provisional application 63/386,784, filed on Dec. 9, 2022.
Claims priority of provisional application 63/384,937, filed on Nov. 23, 2022.
Prior Publication US 2024/0344087 A1, Oct. 17, 2024
Int. Cl. C12N 15/00 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/90 (2006.01)
CPC C12N 15/907 (2013.01) [C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/111 (2013.01); C12N 2310/20 (2017.05)] 18 Claims
 
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).