The Generative Brain

AI powered fight to overcome degenerative brain disorders

No family should face prion disease alone, or without hope.

Creutzfeldt-Jakob disease and other prion diseases move quickly and take people far too soon, and there is still no cure. We are here to change that.

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What happens in prion disease

How prion disease develops in the brain
  1. Normal proteinEveryone makes prion protein. It sits on the surface of cells.
  2. A wrong foldSome of the protein folds into the wrong shape.
  3. It spreads its shapeThe misfolded form causes more protein to misfold.
  4. Build-upMisfolded protein gathers and is hard to clear.
  5. Nerve cells are damagedThis causes the symptoms of the illness.

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Our commitment

We are working toward a way to prevent these diseases and, one day, to cure them. We will keep going until that day comes.

Every family who has lived through this deserves that promise. So we are gathering what the world knows about prion disease and putting the best of AI to work beside the scientists, clinicians and families leading this fight. Our aspiration is to become the leading AI front in that effort, turning scattered knowledge into faster answers.

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Where hope is coming from

Where treatments in development try to step in
  1. The PRNP geneSwitch off or edit the gene that makes the protein.
    • Base editing
    • Epigenetic editing
    • Zinc finger repressors
  2. The RNA messageLower how much protein the cell makes.
    • ION717
    • PrP siRNA
  3. The prion proteinBind the protein or change how cells handle it.
    • PRN100 antibody
    • Small molecules
  4. The brain's responseSupport the cells reacting to the damage.
    • Early ideas

A simplified view: none of these is a proven treatment. ALN-6457 also targets PRNP, but the trial registry does not state its drug type, so it is not placed here.

How treatment research works

A century of prion disease
  1. 1920sCreutzfeldt and Jakob describe the disease that carries their names
  2. 1957Kuru is described in Papua New Guinea
  3. 1982Stanley Prusiner names the prion
  4. 1986BSE is identified in cattle
  5. 1996Variant CJD is reported in the UK
  6. 1997Prusiner receives the Nobel Prize
  7. TodayThe first gene-targeted medicines are in human trials

Key dates from published histories. Sources differ by a year on the first BSE cases.

How we will get there

  1. Bring it togetherEverything known about these diseases, in one place, open to all.
  2. Find the gapsUse AI to connect the evidence and show where progress is stuck.
  3. Connect peopleLink families, scientists, clinicians and funders who can move things forward.
  4. Reach prevention and a cureShorten the road from question to answer, for as long as it takes.

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