Kate Therapeutics is a patient-focused biotechnology company developing AAV-based gene therapies to treat genetically defined muscle disease. We are applying novel technology platforms that directly address the key limitations of current gene therapies, including tissue-specific delivery and gene regulation. These breakthroughs have the potential to improve efficacy and safety of gene therapy and enable the pursuit of a broader set of targets that are otherwise difficult to drug with current technologies.

We have recently published data supporting one of our approaches to improve AAV-mediated gene delivery (Tabebordbar et al., Cell, 2021). Our novel capsids provide superior muscle transduction and liver de-targeting, above and beyond naturally occurring serotypes currently in clinical trials. This may enable more complete tissue transduction at significantly lower viral doses, thus addressing one of the major headwinds facing muscle gene delivery.

Kate Therapeutics is headquartered in San Diego, CA.