> The result isn't saving 3 people but rather making it possible (profitable?) to do the same for anyone with these sorts genetic transcription errors.
The amount of work that goes into making sure that any single treatment isn't going to have unknown side effects is non-trivial. Just because there is a common toolkit doesn't make this safety testing go away. It automates the design of the safety tests somewhat, but doesn't make them any less important. This means that for every... single... anti-sense oligo that's designed, there needs to be significant testing done to make sure that it does what is expected and doesn't have any adverse side effects.
In many cases, it's the first part -- the design of the treatment -- that is rate limiting. The benefit of something like these N-of-1 ASO trials is that you're streamlining the first step in the process. But that doesn't mean you can just skip the other steps. And yes, these steps are expensive.
The amount of work that goes into making sure that any single treatment isn't going to have unknown side effects is non-trivial. Just because there is a common toolkit doesn't make this safety testing go away. It automates the design of the safety tests somewhat, but doesn't make them any less important. This means that for every... single... anti-sense oligo that's designed, there needs to be significant testing done to make sure that it does what is expected and doesn't have any adverse side effects.
In many cases, it's the first part -- the design of the treatment -- that is rate limiting. The benefit of something like these N-of-1 ASO trials is that you're streamlining the first step in the process. But that doesn't mean you can just skip the other steps. And yes, these steps are expensive.