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
Zebrafish line – COMPLETED
- Two models available for drug screening
- $50,000 to $100,000 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 – Soticlestat trial complete
- $1.8 million
- 1.5 years
Phase III Clinical Trial – Marigold trial complete
- $8 million
- 3 years
CDKL5 Clinical consortium – in progress
- ongoing
- NIH Clinical Trial Readiness U01 Grant in progress starting 2021
- $450,000 per year
DATABASE – in progress
- Ongoing
- Critical for determining Therapeutic Endpoints for Trials
- $75,000