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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 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

Skeletal muscle and cardiac promoters used in current gene therapies are limited in their activity and specificity. Kate Therapeutics is applying in vivo massively parallel reporter assay (MRPA) and machine learning to develop novel and compact enhancer and promoter combinations to drive cell type-specific transgene expression.

Reducing Transgene Expression in Off-target Tissues by Leveraging Endogenous miRNA Expression

Kate Therapeutics has identified miRNAs highly expressed in off-target tissues by unbiased miRNA profiling in tissue samples. We reduce expression of the transgene in off target tissues by incorporating the binding site of the identified miRNAs in our therapeutic transgenes.

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.