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公开(公告)号：US20240091378A1
公开(公告)日：2024-03-21
申请号：US18167598
申请日：2023-02-10
Applicant: The Regents of the University of California
Inventor： David V. Schaffer , Leah C. Byrne , Timothy P. Day , John G. Flannery
CPC classification number: A61K48/0041 , C07K7/06 , C07K7/08 , C12N9/22 , C12N15/1082 , C12N15/11 , C12N15/86 , C12N2310/20 , C12N2750/14122 , C12N2750/14142 , C12N2750/14145 , C12N2750/14171 , C12N2800/80
Abstract: The present disclosure provides recombinant adeno-associated virus (AAV) virions with altered capsid protein, where the recombinant AAV (rAAV) virions exhibit greater ability to cross barriers between intravitreal fluid and retinal cells, and thus greater infectivity of a retinal cell compared to wild-type AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.
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公开(公告)号：US11931426B2
公开(公告)日：2024-03-19
申请号：US18356029
申请日：2023-07-20
Applicant: SNIPR Technologies Limited
Inventor： Jasper Clube
IPC: C12N15/63 , A61K35/74 , A61K48/00 , A61P31/04 , C12N9/22 , C12N15/90 , C12P19/34 , C12N15/70 , C12N15/74
CPC classification number: A61K48/0066 , A61K35/74 , A61P31/04 , C12N9/22 , C12N15/63 , C12N15/905 , C12N15/907 , C12P19/34 , C12N15/70 , C12N15/74 , C12N15/902 , C12N2310/20 , C12N2800/80
Abstract: The invention relates to retrieving or modifying target nucleic acids, such as host cell chromosomal DNA, by homologous recombination with vectors that have been cut to provide recombinogenic nucleic acid strands in situ. In some aspects, a marker sequence is created by the method of the invention, wherein the marker sequence is in the product of the method but not present in the starting nucleic acid strands of the method.
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公开(公告)号：US20240084333A1
公开(公告)日：2024-03-14
申请号：US18467428
申请日：2023-09-14
Applicant: Flagship Pioneering Innovations VI, LLC
Inventor： Stephen Hoyt Cleaver , Barrett Ethan Steinberg , Jacob Rosenblum Rubens , Robert James Citorik , William Edward Salomon , Zi Jun Wang
CPC classification number: C12N15/907 , C12N9/1276 , C12N9/22 , C12N15/11 , C12N2310/20 , C12N2800/80
Abstract: Methods and compositions for modulating a target genome are disclosed.
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公开(公告)号：US11926843B2
公开(公告)日：2024-03-12
申请号：US17478374
申请日：2021-09-17
Applicant: LifeEDIT Therapeutics, Inc.
Inventor： Alexandra Briner Crawley , Rodolphe Barrangou , Tyson D. Bowen , Michael Coyle , Tedd D. Elich
CPC classification number: C12N15/907 , C12N9/22 , C12N15/11 , C12N15/902 , C12N2310/20 , C12N2800/80
Abstract: Compositions and methods for binding to a target sequence of interest are provided. The compositions find use in cleaving or modifying a target sequence of interest, visualization of a target sequence of interest, and modifying the expression of a sequence of interest. Compositions comprise RNA-guided nuclease polypeptides, CRISPR RNAs, trans-activating CRISPR RNAs, guide RNAs, and nucleic acid molecules encoding the same. Vectors and host cells comprising the nucleic acid molecules are also provided. Further provided are CRISPR systems for binding a target sequence of interest, wherein the CRISPR system comprises an RNA-guided nuclease polypeptide and one or more guide RNAs.
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公开(公告)号：US11920156B2
公开(公告)日：2024-03-05
申请号：US16484813
申请日：2018-02-08
Applicant: Indapta Therapeutics, Inc.
Inventor： Guy Dipierro
IPC: C12N5/0783 , A61K35/17 , A61P35/00 , C07K14/725 , C07K14/735 , C07K16/28 , C12N9/22 , C12N15/11 , C12N15/113
CPC classification number: C12N5/0646 , A61K35/17 , A61P35/00 , C07K14/7051 , C07K14/70535 , C07K16/2887 , C12N9/22 , C12N15/11 , C12N15/1138 , C07K2317/24 , C07K2317/732 , C07K2317/76 , C07K2319/30 , C12N2310/14 , C12N2310/20 , C12N2510/00 , C12N2800/80
Abstract: The present invention provides engineered Natural Killer (NK) cells and methods of producing engineered NK cells. The engineered NK cells and compositions containing the engineered NK cells are useful for treating diseases such as cancer.
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公开(公告)号：US11920148B2
公开(公告)日：2024-03-05
申请号：US16487300
申请日：2018-02-21
Applicant: CRISPR THERAPEUTICS AG
Inventor： Ante Sven Lundberg , Samarth Kulkarni , Lawrence Klein , Hari Kumar Padmanabhan , Yvonne Sarah Aratyn
CPC classification number: C12N15/86 , C12N7/00 , C12N9/22 , C12N15/111 , C12N2310/20 , C12N2750/14143 , C12N2800/80
Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 gene in a cell by genome editing.
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公开(公告)号：US11920128B2
公开(公告)日：2024-03-05
申请号：US16516996
申请日：2019-07-19
Applicant: Kymab Limited
Inventor： Allan Bradley , Hanif Ali , E-Chiang Lee
IPC: A61K48/00 , C07K16/00 , C12N5/0781 , C12N15/10 , C12N15/90
CPC classification number: C12N15/102 , A01K67/0278 , C07K16/00 , C12N5/0635 , C12N15/907 , A01K2207/15 , A01K2217/052 , A01K2217/072 , A01K2227/105 , A01K2267/01 , C07K2317/14 , C07K2317/20 , C07K2317/24 , C07K2317/52 , C07K2317/56 , C12N2510/04 , C12N2800/80
Abstract: The invention relates to an approach for introducing one or more desired insertions and/or deletions of known sizes into one or more predefined locations in a nucleic acid (eg, in a cell or organism genome). They developed techniques to do this either in a sequential fashion or by inserting a discrete DNA fragment of defined size into the genome precisely in a predefined location or carrying out a discrete deletion of a defined size at a precise location. The technique is based on the observation that DNA single-stranded breaks are preferentially repaired through the HDR pathway, and this reduces the chances of indels (eg, produced by NHEJ) in the present invention and thus is more efficient than prior art techniques. The invention also provides sequential insertion and/or deletions using single- or double-stranded DNA cutting.
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公开(公告)号：US20240068048A1
公开(公告)日：2024-02-29
申请号：US18280103
申请日：2022-03-02
Applicant: Logomix, Inc.
Inventor： Yasunori AIZAWA
IPC: C12Q1/6888 , C12N9/22 , C12N15/11 , C12N15/90 , C12Q1/6869
CPC classification number: C12Q1/6888 , C12N9/22 , C12N15/11 , C12N15/907 , C12Q1/6869 , C12N2310/20 , C12N2800/80 , C12N2800/90 , C12Q2600/124 , C12Q2600/156
Abstract: The present invention provides a method of in vitro modification of genomic DNA in a eukaryotic cell and a method of detecting modification. The present invention can involve, for example, introducing a nucleotide sequence having 50% or more sequence identity to any one of nucleotide sequences of retrotransposons of genomic DNA. The present invention can also involve changing an insertion position, to each neighboring sequence, of at least one of nucleotide sequences of retrotransposons. The present invention provides a modified eukaryotic cell obtained by such a method.
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公开(公告)号：US20240052396A1
公开(公告)日：2024-02-15
申请号：US18124212
申请日：2023-03-21
Applicant: Harbinger Health, Inc.
Inventor： Anthony P. Shuber
IPC: C12Q1/6806 , C12N9/22 , C12N15/11
CPC classification number: C12Q1/6806 , C12N9/22 , C12N15/11 , C12N2800/80 , C12N2310/20
Abstract: The invention provides methods of selectively protecting nucleic acids of interest in a sample from damage that occurs during preparative procedures. The methods include binding proteins to ends and to one or more internal regions of a segment of the nucleic acid of interest so that damage to exposed regions of the segment does not lead to degradation of the entire segment.
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公开(公告)号：US11898179B2
公开(公告)日：2024-02-13
申请号：US16492548
申请日：2018-03-09
Applicant: President and Fellows of Harvard College
Inventor： Juan Pablo Maianti , David R. Liu
IPC: C12N9/78 , A61K9/00 , A61K31/7088 , A61K38/46 , C12N9/22 , C12N9/96 , A61K48/00 , C12N15/11 , A61K38/50
CPC classification number: C12N9/78 , A61K9/0029 , A61K9/0053 , A61K31/7088 , A61K38/465 , A61K38/50 , C12N9/22 , C12N9/96 , C12N15/11 , C12Y305/04 , A61K48/00 , C07K2319/00 , C12N2310/20 , C12N2320/31 , C12N2320/32 , C12N2800/80
Abstract: Provided herein are systems, compositions, kits, and methods for the suppression of pain (e.g., chronic pain). Genes encoding ion channels (e.g., SCN9A) responsible for the propagation pain signals in neurons (e.g., DRG neurons) may be edited using a genome editing agent (e.g., a nucleobase editor). In some embodiments, loss-of-function ion channel mutants are generated, leading to pain suppression. In some embodiments, the genome editing agent is administered locally to the site of pain or to the nerves responsible for propagation of the pain signal.
