Too kind, Nick! Can't wait to listen to the podcast

So maybe the same will be true for these n=1 gene editing treatments. Or a new model for development/payment will emerge. Maybe this really will be the "future of medicine", a phrase that came up a lot in articles about KJ.
I have hope, I just don’t know how long it will take us to get there. 12/12

But…I remember back when cell therapies were deemed too complex and expensive to be commercially viable, and now look at all the CAR T cell therapies. (Those still need to be cheaper and less complex, too, but researchers are working on that. Step-by-step.) 11/12

Each therapy would have to be designed, tested, produced fresh for each recipient. Gene therapies and gene-editing therapies on the market now are already staggeringly expensive, and those companies can spread the cost of development over many recipients, not just one. 10/12

Lots of other parents of children with rare genetic disorders would like their kids to have more joy. For now, it’s hard to know how many of them will have an option like KJ. The usual biotech/commercial models won't work well for this. 9/12

Last year, I spoke to researchers who tried to develop a base-editing therapy for a young woman with a devastating genetic illness. www.nature.com/articles/d41...
They worked round the clock but didn't make it in time. Thankfully KJ’s case was different and, so far, he's doing well. 8/12

KJ’s parents decided to take a chance on CRISPR, and the clock started ticking. Gene editing therapies are complicated to design, test, produce. Each day brought added risk that KJ might die or become disabled. 7/12

KJ’s dad said that until then, most discussions about KJ had been about the chance that he might die or be severely disabled. KJ's dad worried his son would suffer.

“You want your kids to have joy, right?” he said. That’s the quote that didn’t make it into our story but keeps me up at night. 6/12