Key facts
- Codirector of the Broad Prion Therapeutic Science program; protocol principal investigator of the PRiSM trial.
- Developed and published CSF PrP assays (ELISA in 225 samples across 13 mutations; mass spectrometry with CV under 15 percent).
- Argued that CSF PrP falls in symptomatic disease, which confounds symptomatic trials and motivates pre-symptomatic trials, and that randomising to a disease endpoint is infeasible.
- Co-author of the ASO survival study (2019), the CHARM and base editing papers and the PRiSM siRNA work.
- Writes cureffi.org, which carried the trial announcements, the PRN100 and ION717 commentary and the tribute to Byron Caughey.
Timeline
- 2025Base editing paper announced.
- 2026cureffi tribute to Byron Caughey.
- 2026cureffi post on the ION717 trial re-opening.
- 2026PRiSM launch announced; community statement.
Institutions
Projects and trials
- Base editing of PRNP
- NIH NeuroNEXT
- Prion Alliance
- Sonia M. Vallabh
- OBSERVE
- PRiSM
- 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
- PRiSM
Research papers
Works with
This page covers public professional work only. To correct it or ask for it to be removed, write to hello@thegenerativebrain.org.
Last checked 15 October 2026
Sources for this page
- NeuroNEXT NN112 PRiSM project page
- Broad Institute news: PRiSM begins enrolling; siRNA study published in Nucleic Acids Research
- Prion protein quantification in human cerebrospinal fluid as a tool for prion disease drug development (Vallabh et al., PNAS 2019)
- Domain-specific quantification of prion protein in CSF by targeted mass spectrometry (Minikel and Vallabh, bioRxiv)
- Rationale for early therapeutic intervention in genetic prion disease (Minikel, Harvard thesis)
- NIH Research Matters: Developing treatments for prion diseases (CHARM)
- Broad Institute: gene editing extends lifespan in mouse model of prion disease (Jan 2025)
- Antisense oligonucleotides extend survival of prion-infected mice (Raymond et al., JCI Insight 2019)
- cureffi: Results in from PRN100 antibody first-in-human dosing (17 Mar 2022)
- cureffi: ION717 trial re-opens with 3rd dosing regimen (17 Mar 2026)
- cureffi: Celebrating Byron Caughey, 1957-2026