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

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

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.

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.