Abstract: Methods and compositions related to a nucleic acid-guided nuclease cell targeting screen are provided. The invention relates to compositions and methods for identifying cell targeting proteins that, when associated with a nucleic acid-guided nuclease (such as Cas9), enables at least the nucleic acid-guided nuclease to be targeted to the surface of a target cell or internalized by a target cell, i.e., a cell targeted by the cell targeting agent.
Type:
Application
Filed:
October 21, 2021
Publication date:
February 3, 2022
Applicant:
Spotlight Therapeutics
Inventors:
Akshay Tambe, Hariharan Jayaram, Steven Strutt
Abstract: 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.
Type:
Application
Filed:
September 21, 2021
Publication date:
January 13, 2022
Applicant:
Spotlight Therapeutics
Inventors:
Hariharan Jayaram, Rina Mepani, Christopher Richardson, Steven Strutt, Eric Estrin, Jillian Astarita, Spencer Wei, Roberto Maldonado
Abstract: 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 antigen binding polypeptide that specifically binds to an extracellular cell membrane-bound molecule, and a site-directed modifying polypeptide that recognizes a nucleic acid sequence. The antigen binding polypeptide and the site-directed modifying polypeptide are stably associated such that the site-directed modifying polypeptide can be internalized into a cell displaying the extracellular cell membrane-bound molecule.
Type:
Application
Filed:
September 21, 2021
Publication date:
January 6, 2022
Applicant:
Spotlight Therapeutics
Inventors:
Hariharan JAYARAM, Eric ESTRIN, Jillian ASTARITA