This is THE hardest problem to solve in a biotech start-up. In the mitochondrial field, companies are going through orphan-drug designation and fast-track appr

This is THE hardest problem to solve in a biotech start-up. In the mitochondrial field, companies are going through orphan-drug designation and fast-track approval processes. This is probably to only advantage researching for niche - rare - diseases: You can save lots of money/time to get the drug in the market. If you can then find some off-label use for you drug, much better.