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：US20240100185A1

公开(公告)日：2024-03-28

申请号：US18255182

申请日：2021-12-02

Applicant: Scribe Therapeutics Inc.

Inventor： Susan TOM ,  Oleh KRUPA ,  Benjamin OAKES ,  Sean HIGGINS ,  Sarah DENNY ,  Brett T. STAAHL ,  Isabel COLIN ,  Maroof ADIL

IPC: A61K48/00 ,  A61P25/16 ,  C12N9/22 ,  C12N15/113 ,  C12N15/90

CPC classification number: A61K48/005 ,  A61P25/16 ,  C12N9/22 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/20 ,  C12N2800/80

Abstract: Provided herein are Class 2 Type V CRISPR systems comprising CRISPR-Cas polypeptides, (e.g., CasX:gRNA systems comprising CasX polypeptides), guide nucleic acids (gRNA), and optionally donor template nucleic acids, useful in the modification of a PTBP1 gene. The systems are also useful in methods for reprogramming certain eukaryotic cells into functional neurons by the knocking down or knocking out the PTBP1 gene in those cells. Also provided are methods of using such systems in methods of treatment of a subject with a PTBP1-related disease.

公开(公告)号：US20240033377A1

公开(公告)日：2024-02-01

申请号：US18266076

申请日：2021-12-09

Applicant: Scribe Therapeutics Inc.

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

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/86 ,  C12N15/11

CPC classification number: A61K48/005 ,  C12N9/22 ,  C12N15/86 ,  C12N15/11 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2750/14151

Abstract: Provided herein polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. The polynucleotides encode for CRISPR proteins, gRNA, and ancillary components of AAV vectors useful in the modification of target nucleic acids. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the target nucleic acid of a gene. Also provided are methods of using such AAV vectors to modify cells having such mutations.

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：US20240124537A1

公开(公告)日：2024-04-18

申请号：US18516722

申请日：2023-11-21

Applicant: Scribe Therapeutics Inc.

Inventor： Jason FERNANDES ,  Sean HIGGINS ,  Sarah DENNY ,  Ross WHITE ,  Emeric Jean Marius CHARLES ,  Addison WRIGHT ,  Benjamin DEMAREE ,  Manuel MOHR ,  Wenyuan ZHOU ,  Benjamin OAKES

IPC: C07K14/47 ,  C12N9/10 ,  C12N15/113

CPC classification number: C07K14/4703 ,  C12N9/1007 ,  C12N15/113 ,  C07K2319/09 ,  C07K2319/81 ,  C12N2310/20 ,  C12N2310/531

Abstract: Provided herein are gene repressor systems comprising fusion proteins, such as fusion proteins comprising a DNA binding domain such as a TALE, zinc finger or catalytically-dead CRISPR protein and guide nucleic acid (gRNA), which are useful in the repression of a proprotein convertase subtilisin/kexin Type 9 (PCSK9) gene. Also provided are methods of using such systems to repress transcription of PCSK9.

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：US20230054437A1

公开(公告)日：2023-02-23

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 ,  C12N15/113 ,  C12N15/85

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：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.

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：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.