Sarepta Therapeutics

well, I think she became a model of the like philanthropic disease foundation, patient advocacy, because, you know, we all know John Crowley sport story, but I think Kathy juicy is similar in that regard.

And I think before you'd see a lot of these, um, and I, yeah, I worked at, uh, CEO Sarepta.

So do Shannon rare disease, a lot of these organizations, they would focus on finding a cure and donating for basic research, um, But it was Kathy and others like the CF Foundation, others said, no, we need drugs like we need to invest to help them get over that valley of death, to get something really translated, translated to something that will work in patients.

And she that was a mandate for her.

The next day, he used another fake Twitter account to cause market panic, this time impersonating Citron Research, another short selling firm.

Using the same playbook, his tweets claimed that Sarepta Therapeutics was also under investigation.

This time, he was only able to cause a 16% drop in the stock price.

And since he'd only made $9 the day before, he went all in this time.

We'll also recap the deals of the past week, which have been plentiful and included Novartis' nearly eight billion dollar bet on mRNA drugs with China's Abogen Biosciences.

Then we'll have an update for you on Sarepta Therapeutics.

That is the embattled biotech we talked a lot about over the course of last year.

It is not out of the woods just yet, but things do seem to be trending in the right direction for the biotech.

[laughs] Exactly.

All right, so next up is a story we've sort of been waiting for, and it could've gone one way or the other.

Fortunately for Sarepta, things are looking up, at least according to Jefferies analysts.

So rewind to the summer of last year.

And I wanna say, I, I think, Jason, we might have...

When I was at Sarepta, I know we were looking at Rana.

Yeah.

And, and I have to say, you know, fast-forward, never thought that would trade for billions, right? [laughs] So we'll-

And in a lot of these diseases that are being targeted, they're, you know, they're frankly just hard studies to do, even if you are effectively replacing protein.

And I think, you know, we've seen a lot of data to just kind of say, "We don't e- we don't even know, even when you're, when you do show protein production, um, you know, is it, is it resulting in a clinical benefit or is it not resulting in a clinical benefit?" And obviously, like, Sarepta is sort of the, you know, hallmark example of this, where it's making microdystrophin, um, to a meaningful amount, but, um, you know, has not sh- demonstrated efficacy on, you know, prospective endpoints in, in pivotal studies, right? So, so I think, I think that's-- there's a lot of question marks as to, um, you know, where are we in, in the technology.

I think there's no question for, like, hemophilia B, Beksa, like definitely works, right? Like if you need to make factor B in, um, blood, you can do it pretty easily.

It's not-- They wind up not being commercially viable because there's so many good ways, um, um, to treat that.

Novartis has a, a trial, uh, that's also testing a pill.

Um, there's Alnylam, Veko, Sarepta, Wave, and Exo- ExoRNA.

Our...

How many was that? Five or six.

It's the way he likes it.

Sarepta has the only drive for DMD, unfortunately.

Is he going to buy GTA 6? I think we're all going to be playing GTA 6 when it comes out.

We're going to have Playstations all over this place.

Yeah.

Well, and I just, my own experience at Sarepta as a genetic technology, right? The reason a lot of people pursue DMD was because it was the first gene that we understood could be manipulated through exon skipping.

And I actually moved the company from Seattle to the Boston area because there was so much more of a rich heritage of neuroscience and applied.

I mean, if you look at Genzyme and Biogen, right, those are two of the big pillars, right? Um, you know, of the Boston ecosystem and they were rooted in success in neuroscience.

So Jeff Jonas was the CEO when you were the CBO.

And I remember after it went public, people would do the calculation of the value of the company per the number of patients that you had and the what nobody can deny when you stop epileptic seizures from happening in a pediatric.

And I would argue that that really fueled, you know, I remember when I was at Sarepta and there was some argument that if you develop a less, you know, less superior drug, it's going to quell R and D and the exact opposite happened, right? You ended up seeing 50 DMD programs because the oligonucleotide that wasn't the best technology out there, right.

Got approved.