Platform Technologies » Cargo Platform
We are Focused on Delivering Life-Changing Therapies to Patients
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.
