公开(公告)号：US11976277B2

公开(公告)日：2024-05-07

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: C12N15/113 ,  A61K9/00 ,  A61K9/50 ,  C07K14/005 ,  C12N9/22 ,  C12N15/11

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：US12163125B2

公开(公告)日：2024-12-10

申请号：US17572208

申请日：2022-01-10

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：US12084692B2

公开(公告)日：2024-09-10

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

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

CPC classification number: C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/20 ,  C12N2740/15043

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.

公开(公告)号：US11613742B2

公开(公告)日：2023-03-28

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 ,  C12N15/86 ,  C12N15/90 ,  A61P25/28 ,  C12N9/06 ,  C12N15/10 ,  C12N15/113

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：US11560555B2

公开(公告)日：2023-01-24

申请号：US17533997

申请日：2021-11-23

Applicant: Scribe Therapeutics Inc.

Inventor： Benjamin Oakes ,  Sean Higgins ,  Hannah Spinner ,  Sarah Denny ,  Brett T. Staahl ,  Kian Taylor ,  Katherine Baney ,  Isabel Colin ,  Maroof Adil

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

Abstract: Provided herein are engineered proteins comprising a RuvC DNA cleavage domain comprising one or more amino acid modifications, and one or more improved characteristics, relative to a naturally occurring RuvC domain. Also provided are gene editing systems comprising engineered proteins, and methods for use thereof.

公开(公告)号：US11535835B1

公开(公告)日：2022-12-27

申请号：US17483681

申请日：2021-09-23

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 ,  A61K48/00 ,  A61P27/02 ,  A61K38/00

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.