	US 12,263,227 B2
	Optimized mRNA encoding CAS9 for use in LNPs
Christopher J. Cheng, San Francisco, CA (US); Andrew Scharenberg, San Francisco, CA (US); Kui Wang, San Francisco, CA (US); and Shailendra Sane, San Francisco, CA (US)
Assigned to CRISPR THERAPEUTICS AG, Zug (CH); and BAYER HEALTHCARE LLC, Whippany, NJ (US)
Appl. No. 17/298,114
Filed by CRISPR THERAPEUTICS AG, Zug (CH); and BAYER HEALTHCARE LLC, Whippany, NJ (US)
PCT Filed Nov. 26, 2019, PCT No. PCT/US2019/063456 § 371(c)(1), (2) Date May 28, 2021, PCT Pub. No. WO2020/112908, PCT Pub. Date Jun. 4, 2020.
Claims priority of provisional application 62/772,278, filed on Nov. 28, 2018.
Prior Publication US 2022/0047723 A1, Feb. 17, 2022
Int. Cl. A61K 48/00 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/88 (2006.01)

CPC A61K 48/0025 (2013.01) [C12N 9/22 (2013.01); C12N 15/111 (2013.01); C12N 15/88 (2013.01); C12N 2310/20 (2017.05); C12N 2320/32 (2013.01)]

18 Claims

1. A lipid-based nanoparticle (LNP) composition comprising: a nucleic acid molecule comprising a nucleotide sequence encoding a synthetic RNA-guided endonuclease; and one or more lipid moieties selected from the group consisting of amino lipids, ionizable lipids, neutral lipids, PEG lipids, helper lipids, and cholesterol or cholesterol derivatives, wherein the nucleic acid molecule is 3.8 kb or less in length, and wherein the synthetic RNA-guided endonuclease comprises amino acid sequence of SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, SEQ ID NO 5, or a sequence having at least 95% sequence identity to SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, SEQ ID NO: 4, or SEQ ID NO 5.