>We were able to deliver these treatments to the children in our ongoing clinical trials thanks only to funding from a generous family whose own child is a participant.
We probably shouldn't put the main focus on rare disease but on genetic manipulation in general. It would be much more valuable to be able to cure arbitrary genetic defects than just the ones that are somewhere between uncommon and extant.
I mean, this has definitely happened in the past; study of various rare genetic diseases has elucidated and illuminated the processes by which diseases occur at a molecular level, and often have utility far outside the original rare disease.
As to whether that means it's an effective payoff if you put more money in rare diseases (and therefore less into non-rare diseases), I can't say, so instead, I have a portfolio with a small but not tiny amount for the collection of all rare diseases.
It's weird to say 'only' since I assume that a lot more went into this effort other than funding. There are a lot of initiatives that have a lot of funding, but little success. Either way, this result is certainly better than nothing, or waiting years/decades for public funding.
'Only' implies that the funding was necessary, not that the funding was fully responsible. Also consider that 'only' was said by the researchers. From their pov the effort is a given, because that is what they control, while the funding is an anomalous outside factor.
It’s just tough because resources are not unlimited. You’ll save many more lives focusing on new antibiotics and then you will on Tay-Sachs. I don’t think there’s enough researchers in the world to focus on every single rare disease.