Key facts
- Codirector of Broad's Prion Therapeutic Science program with Eric Minikel; senior group leader at the Broad.
- Co-leads the program behind the first two PrP-lowering clinical trials the Broad team helped enable (ION717 and PRiSM) and the preclinical editing programs (CHARM, base editing, zinc finger repressors).
- Collaborated with Ionis from 2014 and with the Khvorova lab from 2019 or 2020.
- Funding: NIH grants and donations from the prion community to the Broad and Prion Alliance.
- Co-founded Prion Alliance, which funds studies including OBSERVE and supports participant travel to trial sites.
Timeline
- 2019Collaboration with Khvorova begins (per Broad).
- 2024CHARM published in Science.
- 2025Base editing paper announced.
- 2026PRiSM launch announced.
- 2026PRiSM pre-symptomatic arm announced.
Institutions
Projects and trials
- Base editing of PRNP
- Eric Vallabh Minikel
- Prion Alliance
- OBSERVE
- anle138b oligomer modulator
- Base editing of PRNP
- CHARM epigenetic editor
- Gate Bioscience oral small-molecule Molecular Gate
- PrP-lowering antisense oligonucleotide
- PrP-lowering divalent siRNA
- Splice-switching small molecules and cryptic exon manipulation to lower PrP
- AAV zinc finger repressor of PRNP
Research papers
Works with
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Last checked 15 October 2026
Sources for this page
- Broad Institute: gene editing extends lifespan in mouse model of prion disease (Jan 2025)
- Broad Institute news: PRiSM begins enrolling; siRNA study published in Nucleic Acids Research
- NIH Research Matters: Developing treatments for prion diseases (CHARM)
- Zinc Finger Repressors mediate widespread PRNP lowering in the nonhuman primate brain and profoundly extend survival in prion disease mice (bioRxiv 2025.03.05.636713; Broad single-cell portal summary)
- ASBMB Today: Hope for prion disease (Jul 2025)
- Prion Alliance: First-in-human trial of PrP-siRNA in symptomatic prion disease
- Prion Alliance: Mid-year update 2026