The study, funded by CRISPR Therapeutics, is an early-stage clinical trial with just 15 participants, but it represents a step forward in a bold quest to treat common diseases with a one-time treatment that tweaks people’s genes.
A host of powerful tools, propelled by the discovery of CRISPR gene editing, which won the Nobel Prize for chemistry in 2020, has sparked the dream of “one and done” treatments for a host of ailments.
That’s a Washington Post gift link so it should get everybody here past the paywall.
In the light of this project, I can’t help but dream a bit: Could I someday benefit from a CRISPR edit? (A Professor Miller 2.0 would surely be a great improvement.) You see, I have a particular diplotype edit in mind due to an extremely rare genetic variant which screws up the manufacture of important enzymes—but I’ll take up that issue in another thread once this one has played out.
The link isn’t working for me, but I can speak a bit about “N=15.”
In a Phase I clinical trial the goal is to show the treatment is “safe”, at least compared to other treatment. When the risk is unknown, review board are not inclined to put more patients at risk to unknown hazard than absolutely necessary, thus the small sample size. With N=15 it is likely a one-sided test at p<0.10. Nothing is certain at this stage, but good results here can lead to further trials.
If this treatment passes Phase I trials (and many do not), then it may progress to Phase II, where the goal is to show the treatment is effective at doing what it is supposed to do. A treatment can fail for safety here too.
Phase III trials are larger, and finally get to the question of whether or not the new treatment is better than existing treatments. These trials are expensive, and as treatments improve it becomes progressively more difficult to show a new treatment is better than the old.