Regulatory Consulting to Support Advancement of a Divalent siRNA for Prion Disease

Broad Institute, Inc., Cambridge MA

Investigators

Abstract

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 our PrP-lowering divalent siRNA therapeutic in prion disease. The goal of this administrative supplement is to provide funding for regulatory consultants who can ensure that our IND application is complete, compliant, coherent, and concise. With guidance from individuals with deep knowledge of the FDA review process, we can submit an IND application with the best possible chance of success, hopefully allowing us to bring this promising therapeutic to clinical trials for patients with prion disease.