If this were to evolve into an actual therapy it would involve many, many edits. Probably on the order of several hundred. So yes, I agree no one would pay $2 million for a 0.3% reduction in Diabetes risk.
As far as costs go, I of course agree. But the costs of manufacturing these treatments isn’t going to be that expensive; guideRNAs, lipid nanoparticles or AAVs are in the hundreds of dollars range for a large batch. At scale, you could probably manufacture treatments for a few hundred bucks. The expensive part is the R&D and the graveyard of treatments that didn’t succeed which the successful ones have to pay for.
A big part of those originate with the expense of getting past the FDA approval process. Many drugs don’t even make it to clinical trials because the approval process expense makes them uneconomical.
I’ve thought about it. But that’s a long way down the road. There’s a lot of basic validation work that has to be done before we need to start thinking about clinical trials. So it’s probably not worth expending any effort on that right now.
Well, sure, there may be a more general argument for FDA bureaucracy being too convoluted (though of course there are risks with it being too lax too—no surer way to see the market flooded with snake oil). But that’s general and applies to many therapies, not just genetic ones. Same goes for research costs being the big slice of the pie.
If this were to evolve into an actual therapy it would involve many, many edits. Probably on the order of several hundred. So yes, I agree no one would pay $2 million for a 0.3% reduction in Diabetes risk.
As far as costs go, I of course agree. But the costs of manufacturing these treatments isn’t going to be that expensive; guideRNAs, lipid nanoparticles or AAVs are in the hundreds of dollars range for a large batch. At scale, you could probably manufacture treatments for a few hundred bucks. The expensive part is the R&D and the graveyard of treatments that didn’t succeed which the successful ones have to pay for.
A big part of those originate with the expense of getting past the FDA approval process. Many drugs don’t even make it to clinical trials because the approval process expense makes them uneconomical.
If the treatment is extensive/expensive enough that traveling isn’t a significant additional incursion, you could try https://www.prospera.co/
I’ve thought about it. But that’s a long way down the road. There’s a lot of basic validation work that has to be done before we need to start thinking about clinical trials. So it’s probably not worth expending any effort on that right now.
Well, sure, there may be a more general argument for FDA bureaucracy being too convoluted (though of course there are risks with it being too lax too—no surer way to see the market flooded with snake oil). But that’s general and applies to many therapies, not just genetic ones. Same goes for research costs being the big slice of the pie.
Yes, I would agree. I don’t think this necessary needs to be that expensive, though it probably will be at the start.