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 at the link.
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.
Yong-Hui JiangĀ
MPI
Yale University
Jiangbing Zhou
MPI
Yale University
Elizabeth M Berry-Kravis
MPI
Rush University Medical Center
Michele Spencer-Manzon
Co-I
Yale University
Hui Zhang
Co-I
Yale University
Caroline Hendry
Co-I
Yale University
James McPartland
Co-I
Yale University
Julie Wolf
Co-I
Yale University
Nigel Bamford
Co-I
Yale University
Kathleen Cardinale
Co-I
Yale University
James Dziura
Co-I
Yale University
Jennifer Panagoulias
FAST Consultant
Allyson Berent
FAST Consultant