Dyne Therapeutics, Inc.

We're gonna pivot to some new data coming out from various public biotechs.

I'm gonna turn it over to Brian again to discuss some new data from Dyne Therapeutics from the 31st Annual World Muscle Society Conference taking place in Hiroshima, Japan.

And then additional news this week from, um, Mirum, uh, which we just spoke about, which came a couple days after news of their FOP drug approval.

So Brian?

Yeah.

So I'll, I'll start with Dyne.

I think Eric may have some comments on it too.

Um, so they had data on their DM1 program, uh, at, um, uh, World Muscle Society this week.

In fact, that's our next slide, right? Soufflé Therapeutics has announced their trial.

Dyne Therapeutics has announced their trial.

Both Soufflé and Dyne are siRNAs targeting Dux4.

And they're a little bit different than Avidity.

And the fallout didn't stop with Novartis.

A competing biotech, Dyne Therapeutics, which is developing a similar treatment for the same disease using a similar trial measurement, a test of how well patients can relax their hand muscles, saw its own odds of success marked down by at least one Wall Street analyst, purely off the read-through from Novartis' data.

It's a good reminder that even a well-funded $12 billion bet on rare disease science can stumble, and that the choice of clinical trial endpoint really matters.

On a more hopeful note, a smaller company called Brain Child Bio closed a $116 million funding round to keep developing cell therapies for childhood brain cancer.