You seem to have assumed something over here. That,
a) A Start-Up has that type of money
b) They'll be willing to invest in something so risky.
As others have pointed out there are StartUps with the money, but somehow we still don't hear about orphan drugs with potential to make it burst onto the scene. Why not? I think that the second factor is more at play over here. Ultimately it comes down to the cost vs. benefit analysis.
Imagine this you are a VC with a $100 mil. to burn will you support a long and twisted development process of something that might not even work at the end?
You might argue that the benefit is that you might become the up and coming Google of pharma, but try telling that to any smart VC.
This is why we need something more than a profit based industry for something that forms the basis of our society. On one hand people talk about freedom, and especially how it's related to the freedom of the markets, and on the other you trap them with the very failings of their bodies.
Perhaps, I am wrong, but this is something that we all need to think about. What do we value more as a species money or wealth? I wish I knew the answer.