> Presumably, curing a genetic disorder for a very small number of people using existing technology is low risk and low reward.
At the present state of medical understanding, in vivo gene therapy is not low risk, just from a financial perspective, leaving aside the medical risks.
In 2017 the FDA approved Luxterna, the first in vivo gene therapy treatment (it had an original list price of 425,000/eye). It fixed RPE65 associated Retinitis Pigmentosa. This spring J&J announced that their LUMEOS trial had missed its primary endpoint, even though it was basically treating the exact same disease, in the same way, just a different gene (RPGR). But only 22 of the 55 treated patients showed improvements on at least two of their measures, and there was no statistically significant improvement on their primary measure. So this stuff is still very much a financial risk, because we simply don't understand human bodies well enough to predict outcomes in a RCT for gene therapy. And if you have to do a Phase III trial to know whether it works or not, then you are going to be forced to spend a whole lot of money to bring any gene therapy to the market.
At the present state of medical understanding, in vivo gene therapy is not low risk, just from a financial perspective, leaving aside the medical risks.
In 2017 the FDA approved Luxterna, the first in vivo gene therapy treatment (it had an original list price of 425,000/eye). It fixed RPE65 associated Retinitis Pigmentosa. This spring J&J announced that their LUMEOS trial had missed its primary endpoint, even though it was basically treating the exact same disease, in the same way, just a different gene (RPGR). But only 22 of the 55 treated patients showed improvements on at least two of their measures, and there was no statistically significant improvement on their primary measure. So this stuff is still very much a financial risk, because we simply don't understand human bodies well enough to predict outcomes in a RCT for gene therapy. And if you have to do a Phase III trial to know whether it works or not, then you are going to be forced to spend a whole lot of money to bring any gene therapy to the market.