[toast0]

> Presumably, curing a genetic disorder for a very small number of people using existing technology is low risk and low reward.

I don't know if that presumption is reasonable. Yes, low reward; but I suspect the risk/cost for a gene therapy is about the same, regardless of the number of people affected. You still need to do all the pre-human trials, which are still expensive; then you've got to do human trials, which are still expensive ... and if there aren't enough potential patients, you might not even be able to run a reasonable trial. (although the article describes a situation of a single patient treatment, you obviously can't run a human trial of that). IMHO, that makes it higher risk than targeting something that affects many more people.

[anon84873628]

Perhaps in the future we will have regulation that allows, let's call it, "experimental" genetic therapy for diseases with a very small patient population, similar to what is already allowed for certain terminally ill patients. That could reduce the economic barriers to treating these groups.