Noted and agreed on R&D costs (especially failed candidates). > there's just about 200k people diagnosed with some form of hemophilia of which only a fraction

Noted and agreed on R&D costs (especially failed candidates). > there's just about 200k people diagnosed with some form of hemophilia of which only a fraction has the funds or the insurance to obtain that kind of treatment in the first place (both the current and the new one) This was actually the crux of the discussion where I learned about the $50k figure. There are a lot of hemophiliacs in countries that spend, on average, just a few hundred dollars per capita each year on health care. Even if Hemgenix went royalty-free right away and no one ever turned a dollar of profit on it, gene-therapy cures are still inaccessible to the bulk of people the World Federation of Hemophilia represents. $3.5 million would be like landing on the moon, and $0.05 million would be like a few orbits at ISS height, but they're both astronomical. They're equally out of reach for most people with severe hemophilia.