Explore the pipeline

See where each treatment idea stands and which studies are running. No treatment has been shown to stop prion disease. This is information about research, and it says nothing about whether a study suits any person.

Where treatment ideas stand

  1. 13Laboratory and animal work
  2. 4First studies in people
  3. 2Later trials
  4. 0Approved
  • EfavirenzPhase 3 trial in Beijing, listed as recruiting since April 2026. Rests on mouse studies. Checked 2026-11-01.
  • Doxycycline in FFI carriersTen year study in one Italian family. Results not found in published sources. Checked 2026-10-15.
  • ION717Phase 1/2a PrProfile trial, given by lumbar puncture. No efficacy data are public. Checked 2026-10-16.
  • PrP siRNAPhase 1 PRiSM trial, one lumbar puncture dose. Checked 2026-10-16.
  • ALN-6457Phase 1/2 trial registered. Targets PRNP; the registry does not state its drug type. Checked 2026-10-16.
  • PRN100 antibodyGiven to six people as individual treatment, outside a trial. No disease was halted. Checked 2026-10-16.
  • Zinc finger repressor ST-506Preclinical. A 3 month safety study in animals is reported. Checked 2026-10-15.
  • Base editingMice only so far. Checked 2026-10-16.
  • CHARM epigenetic editorMice only so far. Checked 2026-10-16.
  • ADAM8 gene therapyPreclinical, from unpublished slides. Checked 2026-10-16.
  • Gate Bioscience pillCompany and conference data in mice. No peer-reviewed paper found. Checked 2026-10-16.
  • KDC203 small moleculeCells and brain slices. Reaching the brain is the open problem. Checked 2026-10-16.
  • PPI-FIT compoundsPreclinical drug discovery. Checked 2026-10-16.
  • Zinc porphyrin compoundPreclinical. Checked 2026-10-16.
  • Dominant-negative gene therapyMice, from a grantee report that is not peer reviewed. Checked 2026-10-16.
  • NSC500EU orphan designation in 2017. No trial registered. Checked 2026-10-17.
  • Serpin inhibitionEarly research. Checked 2026-10-21.
  • GPNMBTissue and cell models. No drug exists yet. Checked 2026-10-16.
  • Glial and inflammation targetsEarly ideas from mouse work. Checked 2026-10-16.

Older medicines including quinacrine, doxycycline, flupirtine and pentosan were tried in people with symptoms, and none extended survival. See repurposed medicines. Stages are simplified by us from each Atlas record.

Trials and studies

  • Interventional, current. Phase 1PRiSMOne dose of PrP siRNA in people with symptoms. A pre-symptomatic arm was announced for October 2026.Checked 2026-10-15
  • Interventional, current. Phase 1/2aPrProfileION717 against placebo. The record lists it as active and not recruiting.Checked 2026-10-15
  • Interventional, current. Phase 1/2ALN-6457Not yet recruiting when last checked. Sites and eligibility were not yet public.Checked 2026-10-16
  • Interventional, current. Phase 3Efavirenz in CJDBeijing. Registered as recruiting, with 246 participants planned.Checked 2026-11-01
  • For people at risk. ObservationalOBSERVEBiomarkers in people at risk of genetic prion disease.Checked 2026-10-16
  • For people at risk. ObservationalPROSPECTUS registry and natural history study for people at genetic risk.Checked 2026-10-16
  • For people at risk. ObservationalNational Prion Monitoring CohortUK cohort of patients and people at high risk.Checked 2026-10-16
  • For people at risk. ObservationalE200K natural historyTel Aviv study of patients and their first-degree relatives.Checked 2026-10-16
  • Finished or reported. FinishedQuinacrine, UCSFNo effect on survival.Checked 2026-11-01
  • Finished or reported. Reported 2009 and 2013Quinacrine, PRION-1 and othersNone showed a survival benefit.Checked 2026-10-16
  • Finished or reported. Reported 2014Doxycycline in CJDNo survival benefit. Stopped early.Checked 2026-10-16
  • Finished or reported. Reported 2004FlupirtineSlower decline on a thinking test, no survival effect.Checked 2026-10-16
  • Finished or reported. Results not publishedDoxycycline in FFI carriersStarted 2011 for ten years.Checked 2026-10-16

Status changes quickly. Check the study registry, and ask the treating neurologist before relying on anything here.

Last checked 1 November 2026