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

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Air date: Wednesday, December 3, 2025, 2:00:00 PM
Time displayed is Eastern Time, Washington DC Local
Views: Total views: 367 (343 Live, 24 On-demand)
Category: WALS - Wednesday Afternoon Lectures
Runtime: 01:10:07
Description: 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 the pros and cons of oligo versus gene therapy for prion disease; regulatory feedback received for both programs; and the central clinical challenge in this rapidly progressive dementia – treating preventively. We will share our work, shaped by FDA input, across the domains of biomarker development, natural history and preclinical studies to enable preventive trials in healthy individuals with known high-penetrance PRNP variants. We will further describe how the mandate to extend healthy life, rather than just illness – one that is shared broadly across the field of neurodegeneration – guides our plans and mission. This talk will be delivered from a patient-scientist perspective as Sonia Vallabh is both a prion disease researcher and herself a carrier of a high-penetrance pathogenic PRNP variant causal for genetic prion disease.

For more information go to https://oir.nih.gov/wals
Author: Sonia Vallabh, Ph.D.
 
CIT Live ID: 57108
Permanent link: https://videocast.nih.gov/watch=57108

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