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

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/85 ,  C12N15/86 ,  C12N15/90

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.

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：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

IPC: C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  C12N15/86

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.

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.

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

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

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.

公开(公告)号：US20250011756A1

公开(公告)日：2025-01-09

申请号：US18663845

申请日：2024-05-14

Applicant: Scribe Therapeutics Inc.

Inventor： Gayathri VIJAYAKUMAR ,  Sean HIGGINS ,  Isabel COLIN ,  Sarah DENNY ,  Brett T. STAAHL ,  Benjamin OAKES ,  Angus SIDORE ,  Suraj MAKHIJA

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/11 ,  C12N15/113 ,  C12N15/85 ,  C12N15/86 ,  C12N15/90

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.

公开(公告)号：US20240360474A1

公开(公告)日：2024-10-31

申请号：US18608127

申请日：2024-03-18

Applicant: Scribe Therapeutics Inc.

Inventor： Katherine BANEY ,  Isabel COLIN ,  Cécile FORTUNY ,  Sean HIGGINS ,  Suraj MAKHIJA ,  Brett T. STAAHL ,  Maroof ADIL ,  Benjamin OAKES ,  Addison WRIGHT ,  Angus SIDORE ,  Manuel MOHR ,  Sarah DENNY

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

CPC classification number: C12N15/86 ,  C12N9/22 ,  C12N15/111 ,  C07K2319/09 ,  C12N2310/14 ,  C12N2310/20 ,  C12N2310/531 ,  C12N2750/14122 ,  C12N2750/14123 ,  C12N2750/14143 ,  C12N2750/14152

Abstract: Provided herein are compositions and methods for use of self-inactivating recombinant vectors (SIRV) encoding Class 2 Type V and guide ribonucleic acid (gRNA) sequences useful for nucleic acid sequence editing, and including self-inactivating components. The SIRV may be delivered to cells as part of an AAV vector to target a gene of interest.

公开(公告)号：US20230183691A1

公开(公告)日：2023-06-15

申请号：US18164138

申请日：2023-02-03

Applicant: Scribe Therapeutics Inc.

Inventor： Jason FERNANDES ,  Sean HIGGINS ,  Isabel COLIN ,  Hannah SPINNER ,  Matthew GARDNER ,  Trent GOMBERG ,  Gayathri VIJAYAKUMAR ,  Sarah DENNY ,  Brett T. STAAHL ,  Maroof ADIL ,  Benjamin OAKES ,  Angus SIDORE ,  Suraj MAKHIJA

IPC: C12N15/11 ,  A61K9/00 ,  A61K9/50 ,  C07K14/005 ,  C12N9/22

CPC classification number: C12N15/111 ,  A61K9/0019 ,  A61K9/5052 ,  C07K14/005 ,  C12N9/22 ,  C12N2310/16 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2320/32

Abstract: Provided herein are delivery particle systems (XDP) useful for the delivery of payloads of any type. In some embodiments, a XDP particle system with tropism for target cells of interest is used to deliver CRISPR/Cas polypeptides (e.g., CasX proteins) and guide nucleic acids (gNA), for the modification of nucleic acids in target cells. Also provided are methods of making and using such XDP to modify the nucleic acids in such cells.

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.