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Directed Evolution of AAV Capsid Leveraging In Vivo Expression of Transgene RNA

Kate Therapeutics is advancing the DELIVER capsid engineering platform to evolve AAV capsid variants that enable potent and selective delivery to target tissues. DELIVER combines diverse capsid library generation with stringent transcript-based in vivo selection and machine learning to evolve functional capsid variants in any cell type of interest in any in vivo model.

DELIVER has been used to evolve a novel class of MyoAAV-LD (liver de-targeted) muscle-tropic capsids, which transduce both skeletal muscle and cardiac tissue with superior potency and selectivity compared to both naturally occurring AAV serotypes (e.g., AAV9, AAV8, AAVrh74) and other engineered capsids, while actively avoiding the liver. These vectors show conserved transduction potency in mice, non-human primates (NHPs), and human muscle cells. Kate Therapeutics is currently advancing sixth generation MyoAAV-LD capsids that enable dramatic de-targeting of the liver, while effectively transducing skeletal muscle and heart tissues after systemic administration in mice and NHPs, further increasing the safety margin for these vectors.

Advantages of DELIVER Platform

  • Diverse first-round virus library designed based on the capsid 3D structure
  • Stringent mRNA-based selection of capsid variants
  • Selection of potent capsid variants in any cell type of interest
  • Machine learning based design of the second-round capsid library
  • Directed evolution in any desired in vivo model

AAV Capsid Library

New Hope for Patients with Muscle and Heart Disease

Kate Therapeutics is a privately-held company with experienced leadership, a world-class scientific advisory team, and leading investor partners.