Eric Vallabh Minikel

Codirector (with Sonia Vallabh) of the Broad Prion Therapeutic Science program, protocol principal investigator of PRiSM and the lead voice on CSF PrP biomarkers and the regulatory evidence argument for pre-symptomatic trials; writes the public blog cureffi.org.

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

  1. 2025Base editing paper announced.
  2. 2026cureffi tribute to Byron Caughey.
  3. 2026cureffi post on the ION717 trial re-opening.
  4. 2026PRiSM launch announced; community statement.

Institutions

Projects and trials

Research papers

Works with

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Last checked 15 October 2026

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