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：US20240123089A1

公开(公告)日：2024-04-18

申请号：US18516840

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: A61K48/00 ,  C12N9/10 ,  C12N9/22 ,  C12N15/113 ,  C12N15/88 ,  C12N15/90

CPC classification number: A61K48/005 ,  C12N9/1007 ,  C12N9/22 ,  C12N15/1137 ,  C12N15/88 ,  C12N15/907 ,  C12Y201/01037 ,  C07K2319/80 ,  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：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：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.