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

IPC: C12N9/22 ,  A61P25/28 ,  C12N9/06 ,  C12N15/10 ,  C12N15/11 ,  C12N15/113 ,  C12N15/86 ,  C12N15/90

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.

公开(公告)号：US20230167424A1

公开(公告)日：2023-06-01

申请号：US17791130

申请日：2021-01-08

Applicant: Scribe Therapeutics Inc.

Inventor： Benjamin OAKES ,  Sean HIGGINS ,  Hannah SPINNER ,  Sarah DENNY ,  Brett T. STAAHL ,  Kian TAYLOR ,  Katherine BANEY ,  Isabel COLIN ,  Maroof ADIL ,  Cole URNES

IPC: C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  A61P3/06

CPC classification number: C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/907 ,  C12Y304/21061 ,  A61P3/06 ,  C12N2310/20 ,  C12N2800/80 ,  A61K38/00

Abstract: Provided herein are systems comprising Class2, Type V CRISPR polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a PCSK9 gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the PCSK9 gene. Also provided are methods of using such CasX:gNA systems to modify cells having such mutations.

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

IPC: C12N9/22 ,  C12N15/11 ,  C12N15/86

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.

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.

公开(公告)号：US20220348925A1

公开(公告)日：2022-11-03

申请号：US17641426

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: C12N15/113 ,  C12N15/10 ,  C12N9/22 ,  C12N9/06 ,  A61P25/28

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.

公开(公告)号：US20220090036A1

公开(公告)日：2022-03-24

申请号：US17483692

申请日：2021-09-23

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: C12N9/22 ,  C12N15/11 ,  C12N15/90

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.

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

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  A61K35/28 ,  A61K35/545 ,  A61K35/34 ,  A61K35/18

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.

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

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C07K14/705 ,  A61P27/02 ,  C12N15/86

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.

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

IPC: C12N9/22 ,  C12N15/86

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.