Are the documents relating to this FDA (?) approval application public? I'm curious about where the current boundaries lie and how the process works.
With CRISPR, it took a long time to figure out how to reliably edit just the gene you want and acceptably minimize off-target edits, including by delivering the therapeutic to just the organ affected and getting the dose and release right.
The public is understandably leery about experimental medical techniques. If they had killed this newborn child with CRISPR therapy, then it might have set created a backlash delaying translation of this technique for years, possibly decades.
In biomedicine, we’re always looking for therapies that approximate the level of precision control available in software. Unfortunately, it’s never more than an approximation, and our ability to measure and predict the size of that error is always limited. That is why the field moves slowly.
Also do we know that this was CRISPR?
https://www.chop.edu/news/worlds-first-patient-treated-perso...
It lasted for around a year and a half (https://www.youtube.com/watch?v=aoczYXJeMY4) before the effect mostly wore off. He took it orally, so it only affected his intestinal lining, and I presume it didn't effect enough stem cells to get a permanent effect, but it would still be usable as something taken annually, which is still far less often than any medication.
"In a race against time, scientists and doctors across the U.S. developed the first in vivo gene therapy, thanks to decades of medical research."
Look at how the Nixon administration and congress gutted NASA. It took nearly 40 years to crawl out of the hole in the roadmaps that their shortsighted stupidity created. We could have had reusable rockets, aerospike engines and Nuclear Thermal Propulsion in the early 1980s. Instead we got halfway measures like the space shuttle and the ISS that both ate budget but didn't create the required innovation for lower cost to orbit.
gavinray•2h ago
https://www.nejm.org/doi/full/10.1056/NEJMoa2504747
gwern•1h ago
tomhow•58m ago