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公开(公告)号：US20240026386A1
公开(公告)日：2024-01-25
申请号：US18255172
申请日：2021-12-02
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Sean HIGGINS , Sarah DENNY , Brett T. STAAHL , Isabel COLIN , Maroof ADIL , Cole URNES
CPC classification number: C12N15/907 , C12N9/22 , C12N15/11 , C12N15/111 , A61K35/28 , A61K35/545 , A61K35/34 , A61K35/18 , C12N2310/20
Abstract: Provided herein are systems comprising Class 2, Type V CRISPR polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a BCL11A gene. The systems are also useful for the modification of cells in subjects with a hemoglobinopathy-related disease. Also provided are methods of treatment of subjects having a hemoglobinopathy-related disease by administration of the systems or nucleic acids encoding such systems that target the BCL11A gene in such subjects.
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公开(公告)号：US20240026385A1
公开(公告)日：2024-01-25
申请号：US18039858
申请日：2021-12-02
Applicant: Scribe Therapeutics Inc.
Inventor： Gayathri VIJAYAKUMAR , Sean HIGGINS , Isabel COLIN , Sarah DENNY , Brett T. STAAHL , Benjamin OAKES , Angus SIDORE , Suraj MAKHIJA
CPC classification number: C12N15/907 , C12N15/11 , C12N9/22 , C12N15/111 , C12N15/86 , C12N2310/20 , C12N2750/14143 , C12N2320/50
Abstract: Provided herein are engineered Class 2, Type V nucleases and guide RNAs useful for the editing of target nucleic acids. Also provided are methods of making and using such variants to modify nucleic acids.
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公开(公告)号：US20230033866A1
公开(公告)日：2023-02-02
申请号：US17780945
申请日：2020-12-04
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL , Cole URNES , Sean HIGGINS
Abstract: Provided herein are Class 2 Type V CRISPR:gNA systems comprising Class 2 Type V CRISPR polypeptides (e.g. CasX), guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a RHO gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the rhodopsin protein. Also provided are methods of using such systems to modify cells having such mutations and utility in methods of treatment of a subject with a RHO-related disease, such as retinitis pigmentosa.
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公开(公告)号：US20230032369A1
公开(公告)日：2023-02-02
申请号：US17829206
申请日：2022-05-31
Applicant: Scribe Therapeutics, Inc.
Inventor： Benjamin OAKES , Sean HIGGINS , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL , Cole URNES
Abstract: Provided herein are CRISPR:guide systems comprising Class 2 Type V polypeptides (e.g. CasX:gNA systems comprising CasX polypeptides), guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a HTT gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the huntingtin protein. Also provided are methods of using such systems to modify cells having such mutations and utility in methods of treatment of a subject with a HTT-related disease, such as Huntington's disease.
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公开(公告)号：US20220220508A1
公开(公告)日：2022-07-14
申请号：US17542219
申请日：2021-12-03
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Sean HIGGINS , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL
Abstract: Provided herein are engineered CasX systems and components thereof, including variant CasX proteins and variant guide nucleic acids (gNAs). The variant CasX proteins and variant gNAs of the disclosure display at least one improved characteristic when compared to a reference CasX protein or reference gNA of the disclosure. In some instances, the variants have one or more improved CasX ribonucleoprotein complex functions. Also provided are methods of making and using said variants.
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Patent Agency Ranking
公开(公告)号：US20240392271A1
公开(公告)日：2024-11-28
申请号：US18693062
申请日：2022-09-21
Applicant: Scribe Therapeutics Inc.
Inventor： Jason FERNANDES , Ross WHITE , Sean HIGGINS , Sarah DENNY , Benjamin OAKES , Emeric Jean Marius CHARLES
Abstract: The disclosure relates to gene repressor systems comprising catalytically-dead Class 2 CRISPR proteins and one or more transcription repressor domains linked to the catalytically-dead Class 2 CRISPR protein as a fusion protein, as well as a guide ribonucleic acid (gRNA); and methods of making and using same.
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公开(公告)号：US20240309344A1
公开(公告)日：2024-09-19
申请号：US17932798
申请日：2022-09-16
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL , Cole URNES , Sean HIGGINS
CPC classification number: C12N9/22 , C12N15/111 , C12N15/86 , C07K2319/09 , C12N2310/531 , C12N2750/14141
Abstract: Provided herein are Class 2 Type V systems comprising nucleases, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a C9orf72 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations or duplications in the C9orf72 gene. Also provided are methods of using such systems to modify cells having such mutations or duplications.
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公开(公告)号：US20240123089A1
公开(公告)日：2024-04-18
申请号：US18516840
申请日：2023-11-21
Applicant: Scribe Therapeutics Inc.
Inventor： Jason FERNANDES , Sean HIGGINS , Sarah DENNY , Ross WHITE , Emeric Jean Marius CHARLES , Addison WRIGHT , Benjamin DEMAREE , Manuel MOHR , Wenyuan ZHOU , Benjamin OAKES
CPC classification number: A61K48/005 , C12N9/1007 , C12N9/22 , C12N15/1137 , C12N15/88 , C12N15/907 , C12Y201/01037 , C07K2319/80 , C12N2310/20 , C12N2310/531
Abstract: Provided herein are gene repressor systems comprising fusion proteins, such as fusion proteins comprising a DNA binding domain such as a TALE, zinc finger or catalytically-dead CRISPR protein and guide nucleic acid (gRNA), which are useful in the repression of a proprotein convertase subtilisin kexin Type 9 (PCSK9) gene. Also provided are methods of using such systems to repress transcription of PCSK9.
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公开(公告)号：US20230124880A1
公开(公告)日：2023-04-20
申请号：US18058251
申请日：2022-11-22
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Sean HIGGINS , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL
Abstract: Provided herein are reference guide nucleic acid scaffolds and variants of reference guide nucleic acid scaffolds capable of binding one or more engineered proteins comprising a RuvC cleavage domain. In some embodiments, the variants of the reference guide nucleic acid scaffolds comprise at least one modification compared to the reference guide nucleic acid scaffold sequences and exhibit one or more improved characteristics compared to the reference guide nucleic acid scaffolds.
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公开(公告)号：US20230081117A1
公开(公告)日：2023-03-16
申请号：US17641404
申请日：2020-09-09
Applicant: Scribe Therapeutics Inc.
Inventor： Benjamin OAKES , Sean HIGGINS , Hannah SPINNER , Sarah DENNY , Brett T. STAAHL , Kian TAYLOR , Katherine BANEY , Isabel COLIN , Maroof ADIL , Cole URNES
IPC: A61K39/00 , C12N9/22 , C12N15/11 , C07K14/725 , C07K14/705 , C07K14/715 , A61K48/00 , A61K45/06
Abstract: Provided herein are CasX:gNA systems, and compositions and methods relating thereto, the systems comprising CasX proteins, guide nucleic acids (gNAs), and optionally donor template nucleic acids useful for the modification cell genes encoding proteins involved in antigen processing, antigen presentation, antigen recognition, and/or antigen response, as well as methods of producing and using populations of cells comprising these modified genes. In some embodiments, the modified cells further express chimeric antigen receptors (CAR) or engineered T cell receptors (TCR). Such systems are useful for preparing cells for immunotherapy.
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