Roadmap to a Cure

Estimated Timelines and Costs

Mouse Models – COMPLETED

  • Need 3-4 mouse models with reproducibility
  • 1-1.5 yr/ mouse
  • $100,000/ mouse
  • Characterization: $50,000/mouse

iPS Cells – COMPLETED

  • Need 3-4 cell lines with reproducibility
  • Correlation to Mouse lines helpful
  • $100,000 to $300,000
  • 6-8 months per line

Protein Target Identification – in progress

  • Proteomics $50,000
  • Mass spectrometry $30,000
  • 1-2 years

Drug Discovery, Screening
and Development – in progress

  • $2.5 million
  • 2-3 years

Phase I (Drug) Clinical Trial

  • $250,000
  • 2 years

Phase II (Pilot) Clinical Trial

  • $1.8 million
  • 1.5 years

Phase III Clinical Trial

  • $8 million
  • 3 years
  • Natural History Study enrolling now

CDKL5 Clinical consortium – in progress

  • ongoing
  • $150,000 per year

DATABASE – in progress

  • Ongoing
  • Critical for determining Therapeutic Endpoints for Trials
  • $75,000