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公开(公告)号：US20240101984A1
公开(公告)日：2024-03-28
申请号：US18168426
申请日：2023-02-13
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
CPC classification number: C12N9/22 , A61P25/28 , C12N9/003 , C12N15/101 , C12N15/1051 , C12N15/11 , C12N15/1133 , C12N15/86 , C12N15/907 , C12N2310/20 , C12N2750/14143
Abstract: Provided herein are CasX:gNA systems comprising CasX polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a SOD1 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the SOD1 protein or the SOD1 regulatory element. Also provided are methods of using such CasX:gNA systems to modify cells having such mutations and utility in methods of treatment of a subject with a SOD1-related disease.
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公开(公告)号：US20240100185A1
公开(公告)日：2024-03-28
申请号：US18255182
申请日：2021-12-02
Applicant: Scribe Therapeutics Inc.
Inventor： Susan TOM , Oleh KRUPA , Benjamin OAKES , Sean HIGGINS , Sarah DENNY , Brett T. STAAHL , Isabel COLIN , Maroof ADIL
IPC: A61K48/00 , A61P25/16 , C12N9/22 , C12N15/113 , C12N15/90
CPC classification number: A61K48/005 , A61P25/16 , C12N9/22 , C12N15/113 , C12N15/907 , C12N2310/20 , C12N2800/80
Abstract: Provided herein are Class 2 Type V CRISPR systems comprising CRISPR-Cas polypeptides, (e.g., CasX:gRNA systems comprising CasX polypeptides), guide nucleic acids (gRNA), and optionally donor template nucleic acids, useful in the modification of a PTBP1 gene. The systems are also useful in methods for reprogramming certain eukaryotic cells into functional neurons by the knocking down or knocking out the PTBP1 gene in those cells. Also provided are methods of using such systems in methods of treatment of a subject with a PTBP1-related disease.
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公开(公告)号：US20240033377A1
公开(公告)日：2024-02-01
申请号：US18266076
申请日：2021-12-09
Applicant: Scribe Therapeutics Inc.
Inventor： Manuel MOHR , Katherine BANEY , Angus SIDORE , Cécile FORTUNY , Maroof ADIL , Addison WRIGHT , Brett T. STAAHL , Sean HIGGINS , Benjamin OAKES , Suraj MAKHIJA , Sarah DENNY
CPC classification number: A61K48/005 , C12N9/22 , C12N15/86 , C12N15/11 , C12N2310/20 , C12N2750/14143 , C12N2750/14151
Abstract: Provided herein polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. The polynucleotides encode for CRISPR proteins, gRNA, and ancillary components of AAV vectors useful in the modification of target nucleic acids. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the target nucleic acid of a gene. Also provided are methods of using such AAV vectors to modify cells having such mutations.
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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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Patent Agency Ranking
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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公开(公告)号：US20250136962A1
公开(公告)日：2025-05-01
申请号：US18909093
申请日：2024-10-08
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 catalytically dead engineered proteins, e.g., catalytically dead CasX proteins. Also provided herein are systems comprising guide nucleic acids and catalytically dead engineered proteins, and methods for use thereof.
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公开(公告)号：US20240376462A1
公开(公告)日：2024-11-14
申请号：US18789158
申请日：2024-07-30
Applicant: Scribe Therapeutics Inc.
Inventor： Gayathri VIJAYAKUMAR , Sean HIGGINS , Isabel COLIN , Sarah DENNY , Brett T. STAAHL , Benjamin OAKES , Angus SIDORE , Suraj MAKHIJA
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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