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.
Type:
Grant
Filed:
February 3, 2023
Date of Patent:
May 7, 2024
Assignee:
Scribe Therapeutics Inc.
Inventors:
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
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.
Type:
Grant
Filed:
September 23, 2021
Date of Patent:
March 28, 2023
Assignee:
SCRIBE THERAPEUTICS INC.
Inventors:
Benjamin Oakes, Sean Higgins, Hannah Spinner, Sarah Denny, Brett T. Staahl, Kian Taylor, Katherine Baney, Isabel Colin, Maroof Adil, Cole Urnes
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.
Type:
Grant
Filed:
November 23, 2021
Date of Patent:
January 24, 2023
Assignee:
Scribe Therapeutics Inc.
Inventors:
Benjamin Oakes, Sean Higgins, Hannah Spinner, Sarah Denny, Brett T. Staahl, Kian Taylor, Katherine Baney, Isabel Colin, Maroof Adil
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.
Type:
Grant
Filed:
September 23, 2021
Date of Patent:
December 27, 2022
Assignee:
Scribe Therapeutics Inc.
Inventors:
Benjamin Oakes, Hannah Spinner, Sarah Denny, Brett T. Staahl, Kian Taylor, Katherine Baney, Isabel Colin, Maroof Adil, Cole Urnes, Sean Higgins
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.
Type:
Application
Filed:
September 9, 2020
Publication date:
November 3, 2022
Applicant:
Scribe Therapeutics Inc.
Inventors:
Benjamin OAKES, Sean HIGGINS, Hannah SPINNER, Sarah DENNY, Brett T. STAAHL, Kian TAYLOR, Katherine BANEY, Isabel COLIN, Maroof ADIL, Cole URNES
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