Paul Matteis
Biotech equity analyst at Stifel, where he has covered the sector since 2018.
“I always have a hard time getting all that excited about a gene therapy program for a disease that has other good therapeutic alternatives or doesn't have some morbidity and mortality to it. … To me, it's just reaffirming that AAV right now feels like it's a rare disease modality. And going more broadly, develop elsewhere at your own peril is sort of how it feels.”
Biotech Hangout · Health & Longevity · November 2026
Context
The hosts were discussing reports of tumors in patients treated with an AAV gene therapy for SMA. He says the risk-benefit in severe SMA probably still holds but draws a broader conclusion about the modality.
Transcript
…this is a complicated market. I think if these were small molecules that were unscheduled, that would be a very, very big risk, right? But for the other psychedelics that are different mechanisms or modalities, like an LSD or 5-MeO DMT, this seems to probably have little impact, I would think.
…ViCat story in the gene therapy, Zolgensma, gene therapy has just had a tough run of it. But, you know, one of the shiny, you know, only shining stars in gene therapy has been Zolgensma. So, Paul, I'll kick it back to you. What did we learn this week about Zolgensma? And what are your takeaways?
Yeah, I'll be brief here because then we have a few other topics. But essentially, it was reported by a few different media outlets that there have been a handful of reports of tumors in the post-marketing setting in patients who've received Zolgensma. I don't know the veracity of these different sorts of other posts or reports, but there had been a parent of a patient who talked about how Zolgensma had been linked to tumors and Novartis had not given all the information or things like that. So again, I don't know how real that is, but this was in multiple articles covered, covered this topic. But I think as we think about the product and we think about the space and the gene therapy space, one notable detail that was reported here is that a couple of the tumor cases had evidence. Of vector integration, which has always been this, you know, theoretical risk around DNA damage or carcinogeny or things like that with AV. I mean, I think in the context of SMA, right? I mean, it's especially you talking type one, type two. I mean, it's so severe, right? It's hard to believe that something like Zolgensma, which is a tremendous therapy, really has like…
Um, yeah, that's a good uh, good rundown. Um, we do have a couple of uh data previews, data uh, look backs. Um, I'm gonna pass it over to your own. Um, would love to kind of hear your takeaways from the key ash abstracts that were released this week, um, as well as I think we have uh um data coming…
Yeah, and and maybe just to pause last point. So, there's actually two recent approvals from ultragenics: one is GST1A, which is a disease that requires cornstarch sort of throughout the day because patients can otherwise literally sort of crash from hypoglycemia. So, it's called ganglycos that's…
I think the most interesting thing about the Ash
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