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Platform Technologies » Cargo Platform

We are Focused on Delivering Life-Changing Therapies to Patients

Kate Therapeutics is pursuing several approaches to making safer and more effective gene therapy cargoes. Our suite of technologies is intended to enhance regulation of gene therapy cargoes to improve pharmacology and safety.

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.