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：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：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

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

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：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：US20220081681A1

公开(公告)日：2022-03-17

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

Abstract: Provided herein are engineered proteins. In some embodiments, the engineered proteins comprise a RuvC DNA cleavage domain comprising one or more modifications, relative to a naturally occurring domain. In some embodiments, the engineered proteins comprise a chimeric domain, for example a chimeric helical I domain. In some embodiments, the engineered proteins are chimeric proteins, comprising at least two domains, each domain derived from a different source, for example derived from SEQ ID NO: 1 and SEQ ID NO: 2. Also provided are systems comprising these engineered proteins, and methods of making and using said engineered proteins.

公开(公告)号：US20250043262A1

公开(公告)日：2025-02-06

申请号：US18778393

申请日：2024-07-19

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 gene editing pairs comprising an engineered protein, e.g, an engineered CRISPR protein and a guide ribonucleic acid (gRNA) that may provide improved gene editing relative to reference, i.e., wild type sequences. In some embodiments, the engineered protein of the gene editing pair comprises a RuvC cleavage domain with one or more amino acid modifications relative to a reference CRISPR protein RuvC sequence. In some embodiments, a guide ribonucleic acid (gRNA) of the gene editing pair comprises at least one modification in a scaffold stem loop relative to a reference scaffold stem loop sequence.

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：US20220177872A1

公开(公告)日：2022-06-09

申请号：US17542238

申请日：2021-12-03

Applicant: Scribe Therapeutics Inc.

Inventor： Benjamin OAKES ,  Sean HIGGINS ,  Hannah SPINNER ,  Kian TAYLOR ,  Sarah DENNY

IPC: C12N15/10 ,  C12N9/22 ,  C12N15/11 ,  C12N15/63 ,  C12N15/70 ,  C12N15/86

Abstract: Provided herein are methods of developing biomolecule variants (such as proteins, RNA, or DNA) with improved characteristics, for example by developing libraries of variants with alterations to one or more specific monomer locations and screening said libraries for characteristics of interest. These alterations can include deletion, substitution, and insertion, and variants may comprise one alteration or a combination of alterations. Said methods may include further iterative cycles of library construction and evaluation to develop, for example, a biomolecule variant with improved characteristics compared to a reference biomolecule. The methods can also provide information that may be used in the rational design of variants.