Therapeutic Editing to Lower PrP in Prion Disease

PROJECT SUMMARY The overarching goal of the parent grant is to file an Investigational New Drug (IND) application with FDA to enable a first in human trial of PRNP-AAV-CHARM, a PrP-lowering epigenetic editor therapeutic, in prion disease. The goal of this administrative supplement is to provide funding to produce clinical-grade drug product at a scale that will support our first-in-human clinical trial in N=10 prion disease patients. This clinical study will very likely be the first time either CHARM or the BI-hTfRv2 viral vector are tested in humans. If this human proof-of-concept is successful, both technologies stand to be of use against a wide range of other CNS diseases.