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公开(公告)号：US11649444B1
公开(公告)日：2023-05-16
申请号：US17819795
申请日：2022-08-15
Applicant: HuidaGene Therapeutics Co., Ltd.
Inventor： Hainan Zhang , Xiangfeng Kong , Qijia Chen
CPC classification number: C12N9/22 , C12N15/102 , C12N2310/20
Abstract: The present disclosure provides a Cas12i protein (e.g., non-naturally occurring, engineered) comprising an amino acid sequence having at least about 80% (e.g., at least about any of 81%, 82%, 83%, 84%, 85%, 86%, 87%, 88%, 89%, 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, 99%, 99.5%, or 100%) identity to the amino acid sequence as set forth in any one of SEQ ID NOs: 1-10 (preferably, SEQ ID NOs: 1-3 and 6, and more preferably, SEQ ID NO: 1). The present disclosure further provides an engineered, non-naturally occurring CRISPR-Cas12i system comprising: (1) any of the Cas12i proteins described herein or a polynucleotide encoding any of the Cas12i proteins described herein; and (2) a CRISPR RNA (crRNA) or a polynucleotide encoding the crRNA, wherein the crRNA comprises: (i) a spacer capable of hybridizing to a target sequence of a target DNA, and (ii) a Direct Repeat (DR) linked to the spacer and capable of guiding the binding of the Cas12i protein to the crRNA to form a CRISPR-Cas12i complex targeting the target sequence.
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公开(公告)号：US20240011005A1
公开(公告)日：2024-01-11
申请号：US18331431
申请日：2023-06-08
Applicant: HuidaGene Therapeutics Co., Ltd.
Inventor： Xiangfeng Kong , Lecong Wang , Xuqiang Kong
CPC classification number: C12N9/22 , C12N15/86 , C12N2310/20 , C12N2750/00043
Abstract: The disclosure provides Cas12f polypeptides, fusion proteins comprising such Cas12f polypeptides, CRISPR-Cas12f systems comprising such Cas12f polypeptides or fusion proteins, and methods of using the same.
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公开(公告)号：US20230330269A1
公开(公告)日：2023-10-19
申请号：US18047255
申请日：2022-10-17
Applicant: HuidaGene Therapeutics Co., Ltd.
Inventor： Shaoran Wang
CPC classification number: A61K48/0066 , C12N15/86 , A61P27/02 , C12N2750/14143 , C12N2750/14145 , C12N2750/14171 , C12N2750/14122 , C12N2830/50
Abstract: The disclosure described herein provides recombinant adeno-associated virus (rAAV) viral particle comprising an AAV9 serotype capsid and a vector genome encoding an RPE65 (e.g., hRPE65) polypeptide, as well as related compositions and uses thereof (e.g., use to treat Leber's congenital amaurosis 2 (LCA2)).
