Platform Clinical Trials of Genome Editors in Multiple Diseases

In phase 2, the SCGE aims to accelerate the translation of genome editing therapies into the clinic by establishing new regulatory pathways to lay the groundwork for clinical trials that assess the safety and efficacy of promising genome editing therapies to treat multiple diseases. Find the latest publications from this research on our website.

A Non-viral CRISPR-mediated Genome Editing Delivery Platform As A Potential Therapy For Neurogenetic Diseases

Genome editing holds great promise for the treatment of many genetic diseases; however its application in the clinic has been slow due to safety challenges as well as low efficiency of targeting one disease at a time. In this study, we propose a new gene editing platform with the potential to treat hundreds or even thousands of diseases. The platform uses a delivery system that specifically targets neurons in the brain in order to correct genetic mutations that result in neurogenetic disease. As a proof-of-concept, two diseases being targeting in this project are Angelman syndrome (AS) and H1-4 syndrome (HIST1H1E syndrome). We will work with the regulatory agents to prepare for clinical translation in the fist phase and then test the genome editing therapy in patients in the second phase. If successful, the study will create a paradigm shift for genome editing; rapidly expanding the number of neurogenetic diseases treated by in vivo gene editing and accelerating the transition of genome editing technology into clinical applications.

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