Kate Therapeutics’ cargo platform includes both internally generated proprietary technology and a license to discoveries from the University of Florida. The cargo platform provides for enhanced tissue or cellular targeting, or de-targeting, of therapeutic payloads, which aims to further improve the pharmacology and safety of AAV vectors.
Cargo Platform Features
Novel Cell Type Specific Enhancer-Promoter Combination Development Informed by Machine Learning
Current skeletal muscle and cardiac promoters are limited in their activity and specificity. Kate Therapeutics is applying comprehensive transcriptome analysis and machine learning to develop novel enhancer and promoter combinations to drive cell type-specific transgene expression.
Tissue-specific Alternative Splicing to Restrict Globally Expressed Therapeutic-AAV (TARGET-AAV)
Promoters that are active in off-target cell types can lead to suboptimal pharmacology and potential toxicity. Kate Therapeutics is leveraging cell/tissue/disease-specific alternatively spliced exons to control transgene translation where it’s needed, and to inhibit translation in off-target cell types.
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.