Third patient dies from acute liver failure caused by a Sarepta gene therapy

https://www.science.org/content/blog-post/sarepta-sarepta

Thoughts from Derek Lowe (In The Pipeline).

A thread from yesterday about why gene therapy hasn't reached its potential: https://news.ycombinator.com/item?id=44573193

A bloomberg archive.ph article about the same topic - https://archive.ph/9qB0t

Whatever policy we pursue, whatever decision we make, is not forever (at a society level).

When people die, we change policy. When people feel like they cannot get the treatment they need, we change policy.

Unfortunately, this is very complicated and emotionally heavy, and it is much easier to set down the burden on someone else's shoulders, in the form of blame.

We want the FDA to do life critical, complicated and contradictory things, so it's easy to create a narrative that blames the FDA.

The other option besides blame is shared responsibility and humility, but it feels like people are not very good at thinking that way right now.

Sarepta's drug uses AAV to deliver the payload. I wonder why they chose AAV instead of lipid nanoparticles.

https://medcitynews.com/2025/07/sarepta-gene-therapy-fatalit...

Their stock is down 90% over the last 6 months, 37% today. That's not good.

Apparently the treatment costs $3.2 million. https://en.wikipedia.org/wiki/Delandistrogene_moxeparvovec

Damn, gene therapy is so promising too

I've been working on a piece about how humans effectively have hardened firmware, and gene therapies need to do A LOT to try to get around the various defenses our bodies evolved. I should probably finish that article...

the paywall really cuts down on the readability of this story. a quick google showed plenty of news stories though, their shareprice dropped 40% on the market today.

I'd be curious what the numbers are for the "good" that this therapy does; is there any way that this therapy is still "worth it" at any scale? but I know little about this area so that's a fairly naive question.