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.
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.
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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.
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.