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AssigneeSpotlight Therapeutics
InventorsHariharan Jayaram, Rina Mepani, Christopher Richardson, Steven Strutt, Eric Estrin, Jillian Astarita, Spencer Wei, Roberto Maldonado
Estimating the term…
Methods and compositions related to intracellular delivery of gene editing proteins are provided. The invention relates to compositions and methods for transporting gene editing polypeptides, such as Cas9 or Cas12, into a cell ex vivo or in vivo. The invention includes a targeted active gene editing (TAGE) agent that includes an extracellular cell membrane binding moiety, e.g., an antigen binding polypeptide, a cell penetrating peptide (CPP), a ligand, or combinations thereof, that specifically binds to an extracellular cell membrane-bound molecule (e.g., a cell surface molecule), and a site-directed modifying polypeptide that recognizes a nucleic acid sequence. The extracellular cell membrane binding moiety (e.g., antigen binding polypeptide, CPP, or ligand) and the site-directed modifying polypeptide are stably associated such that the site-directed modifying polypeptide can be internalized into a cell, such as one displaying an extracellular cell membrane-bound molecule recognized by the extracellular cell membrane binding moiety.
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
A site-directed modifying polypeptide comprising an RNA-guided DNA endonuclease that recognizes a CRISPR sequence and a conjugation moiety that binds to an antibody, an antigen-binding portion of an antibody, or an antibody mimetic that specifically binds to an extracellular cell membrane-bound molecule.
A site-directed modifying polypeptide comprising an RNA-guided DNA endonuclease that recognizes a CRISPR sequence and a conjugation moiety that binds to a ligand that facilitates uptake of a polypeptide to which the ligand is conjugated.
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
A targeted active gene editing (TAGE) agent comprising
The TAGE agent of claim 77, wherein the site-directed modifying polypeptide further comprises at least one nuclear localization signal (NLS).
A method of modifying a nucleic acid sequence within a target cell in a mammalian subject, the method comprising contacting the target cell in the subject with a targeted active gene editing (TAGE) agent comprising an extracellular cell membrane binding moiety, and a site-directed modifying polypeptide that recognizes the nucleic acid sequence within the target cell, such that the nucleic acid sequence of the target cell is modified.
A method of modifying a nucleic acid sequence within a target cell in a mammalian subject, the method comprising locally administering to the subject a targeted active gene editing (TAGE) agent comprising an extracellular cell membrane binding moiety, and a site-directed modifying polypeptide that recognizes the nucleic acid sequence within the target cell, such that the nucleic acid sequence of the target cell is modified.
A method of modifying a nucleic acid sequence within a target mammalian cell, the method comprising contacting the target mammalian cell with a targeted active gene editing (TAGE) agent under conditions in which the TAGE agent is internalized into the target cell, such that the nucleic acid sequence is modified,
Dependencies are inferred from the claim text.