WALS: A Patient-Scientist Lens on How and Why to Move Faster to First in Human

From NIH VideoCast

This talk will describe our lab's efforts to advance to small academic-initiated first-in-human studies both a divalent siRNA therapy for prion disease, and a one-time epigenetic therapy called CHARM (Coupled Histone tail for Autoinhibition Release of Methyltransferase). Both di-siRNA and CHARM have been shown capable of dramatic disease modification in prion-infected mice. This talk will discuss the timelines and central challenges of these two rapidly advancing programs. Topics will include...