The Generative Brain

Broad Institute: Vallabh/Minikel Prion Therapeutic Science program

Broad Institute program led by Sonia Vallabh and Eric Minikel (Prions@Broad, launched 2015; Prion Therapeutic Science program). It develops PrP-lowering drugs (the PRiSM siRNA, with the ION717 antisense program it helped enable) and one-time editing approaches (CHARM, base editing, zinc finger repressors), and runs the regulatory and biomarker work behind pre-symptomatic trials.

Key facts

  • Prions@Broad launched in fall 2015 with BroadIgnite seed funding; after their PhDs in 2019 Vallabh and Minikel founded an independent lab at the Broad and co-direct the Prion Therapeutic Science program.
  • Lab headcount of eight in 2022; funded by NIH grants and by donations to the Broad and to Prion Alliance (founded 2012).
  • Clinical: sponsor of PRiSM (NCT07444580, NIH NeuroNEXT) and a collaborator in the Ionis ION717 program.
  • Preclinical: CHARM epigenetic editor (Science 2024), base editing (Nature Medicine 2025), zinc finger repressors with Sangamo (preprint 2025; program now owned by Lilly) and a small-molecule collaboration with Gate Bioscience since 2021.
  • Publishes its FDA regulatory documents and trial protocols openly on the Vallabh/Minikel lab site and shares news on CureFFI.org.

Timeline

  1. 2015Prions@Broad launched with BroadIgnite seed funding.
  2. 2019Independent lab opened at the Broad.
  3. 2024CHARM published in Science.
  4. 2025Base editing paper announced.
  5. 2026PRiSM trial launched.
Sources for this page