BioCentury This Week

Ep. 352 - A Multipolar Biopharma World; Rare Disease Spotlight

• BioCentury • Season 7 • Episode 352

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Can a strong U.S. biopharma industry be reconciled with the successful emergence of China? And can China be a catalyst of positive change across the global industry, even if this implies some level of rebalancing away from the U.S.? On the latest BioCentury This Week podcast, BioCentury’s analysts discuss a Guest Commentary by McKinsey Senior Partner Emeritus Franck Le Deu, who argues that a multipolar biopharma world in which the U.S. continues to thrive even as China becomes meaningfully stronger can emerge.
The analysts also discuss BioCentury’s latest Rare Disease Spotlight, which focuses on a wave of therapies aiming to activate retinal function in Stargardt disease patients.
Turning to FDA, BioCentury Washington Editor Steve Usdin discusses recent public statements by FDA Commissioner Marty Makary, questions about Vinay Prasad’s future, and the plausible mechanism pathway.

View full story: https://www.biocentury.com/article/658606

 #GlobalBiopharma #ChinaBiotech #RareDisease #StargardtDisease #FDAPolicy 

00:00 - Introduction
02:47 - Multipolar Biopharma World
13:39 - Rare Disease Spotlight
25:15 - Makary Remarks
31:14 - Plausible Mechanism Framework

To submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.

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[AI-generated transcript.]

Jeff Cranmer:

Why a strong US biotech sector can still thrive next to a stronger China, we discuss McKinsey Asia veteran Franck Le Deu vision of a multipolar biopharma world on the latest BioCentury This Week podcast. And in the rare disease spotlight, Stargardt’s first approval is now in sight as next generation aims to restore vision. Plus FDA Commissioner Makary's extraordinary public statements, questions about Vinay Prasad's future, and the Plausible Mechanism Pathway. I'm Jeff Cranmer, host of the BioCentury's This Week podcast, and joining me today are my colleagues.

Simone Fishburn:

Simone Fishburn, Editor in Chief.

Selina Koch:

Selina Koch, Executive Editor.

Tierney Baum:

Tierney Baum, Biopharma Analyst.

Steve Usdin:

And Steve Usdin, Washington Editor.

Jeff Cranmer:

Okay, we are just days away from BioCentury's 5th East-West Summit in Seoul. there are still some delegate seats left if you would like to attend go to BioCenturyEastWest.com. We'll be debuting the BioCentury Lounge, which is a dedicated space designed to connect, recharge, and inspire conversation among industry leaders. It's a subscriber only space. I look forward to checking this out. And East-West, what can I say? It will be March 9th through the 11th. at the Westin in downtown Seoul. And we'll also be doing an excursion to Daejeon, which has been called South Korea's, Silicon Valley, where we'll be hosted by the mayor. So

Simone Fishburn:

I am excited to see you in that lounge, Jeff. Uh. Maybe, uh, may, maybe we can get a mug or two from BioCentury This Week podcast

Jeff Cranmer:

Yeah. I'll try to smuggle a few into my, uh, into my luggage and, uh. I'm also gonna rep my beloved San Francisco Giants gear as I head to Korea, of course, one of our star players, Jung Hoo Lee hails from Korea. He was a star in the Korean Baseball League. Uh, Son of the Wind, was his dad and that got him the nickname, Grandson of the Wind. And of course. Uh, some of our Japanese listeners will remember his dad, from his days playing for the Chunichi Dragons, back in the nineties in the Japanese professional baseball league. But yes, it is baseball season so excited about that, but more excited to talk biotech. And we're gonna start off with a commentary that's out today on BioCentury.com by Franck Le Deu, who led McKinsey in Asia for many, many years. Franck has just joined Nisa Leung's, new firm, Aulis Capital, where he's a venture partner and, uh, longtime listeners of the pod will know that Franck has been on many times, uh, around our conferences and always has some great insights into what's going on in China. So, Selina, you edited Franck's commentary. What's his argument in support of this multipolar biopharma world?

Selina Koch:

Yeah, well this was inspired by his recent participation at, um, a pharma forum, which our colleague, Steve, on this podcast also, attended where he was on the panel on, um, uh, the rise of China, right? And he was said, he was just struck by how quickly the conversation always slides into a, a zero sum sort of framing where, you know, America can only continue to lead in biopharma if China's contained right. And he's saying, okay, well as a somebody from Europe, from neither of those two polls, he just brings a different perspective, which is that he thinks, we really need this, as he calls it, a multipolar world where you have strong centers in different places and people who are savvy enough to understand how to effectively and efficiently use the strengths of different regions to speed up, which what is currently a highly efficient failure prone sort of sector. Um, I know Steve, you were at the forum. I dunno

Steve Usdin:

I, I, I was at the forum and it was, it really was striking. I wrote about it, um, what was it, last week or the week before, I can't remember. And, and I wrote about Franck's comments at the time, and one of the things that also struck me that wasn't said at the Forum but is true, and I wrote about it, is that on the one hand you have multinational pharmaceutical companies, including multinational pharmaceutical companies that are headquartered in the United States, lamenting publicly lamenting, the rise of the Chinese biopharma sector. And at the same time, those very companies are investing billions of dollars in the Chinese biopharma sector. So on the one hand, they're saying that this poses a threat to United States, a threat to the world and so on. And at the other hand what their actions are doing is integrating, China into the global biopharma ecosystem. And Franck basically was saying, you shouldn't think of this as a zero sum game in which, something being developed in China somehow is bad for the world. And he pointed out that a, for example, a lung cancer drug that was example he gave, that is developed in China and works for people in China. It's gonna work for people in the United States. It's gonna work for people in France. And from the perspective of patients growing the pie, creating more new therapies, is what, everyone should want. So one of the other things that he points out in this commentary that's very interesting is he compares, the United States and Europe. He points out that Europe, uh, has a population that's comparable to the United States, actually larger. It has excellent science, but it doesn't have the kind of stature that the United States does in the global innovation ecosystem. And, you know, I, I, I think, go ahead.

Simone Fishburn:

I, I thought it was really interesting, partly 'cause I look obviously a lot at Europe and I I wanted to talk about that and then so a couple of other points you made. So, with Europe he sort of creates this, you know, he talks about something that we talk about a lot and everybody talks about a lot is like the lack of, growth capital in Europe. They've got all the substrate. He draws a line also to the pricing in Europe and he really sort of dangles something tantalizing, which is, you know, if they could move on pricing, maybe they could just dramatically change. That's sort of what I read through from what he said is, you know, that could actually create, more investment in Europe, even by the pharmas, you know, who, who could go there and sort of, it's almost like a, a tying the idea of, drug pricing solutions in Europe to growth capital and to their success. But I think that it's not totally new to talk about that, but he talks about that in the context of this new world. So it used to be just so monopolar with the US.

Steve Usdin:

And I, well, the, the other thing to look at it is, it's somewhat of a cautionary tale also because some of the things that he attributes to Europe losing its position as, um, kind of coequal in drug development to the United States are things that maybe are creeping into, into our lives in the United States. and.

Simone Fishburn:

Right,

Steve Usdin:

If, if, if care isn't taken, then rather than becoming more competitive, rather than taking the Chinese challenge as an opportunity to make life sciences development in the United States and in Europe more efficient and better, we might actually be going the wrong direction. And at that forum, Scott Gottlieb, the former FDA Commissioner, who's also a, a VC, and he is on the board of various life sciences companies. He said, look, in some ways the United States is stepping on the brakes while China is stepping on the gas.

Simone Fishburn:

Yeah, I think that's so true. So I just wanted to go through a couple of the things that he talks about that could disrupt the status quo, which, you know, again, moving away from this China versus US polarity, and he talked also at the level of innovation becoming more abundant. So he talks about it as me, better me faster, becoming the norm, right? True breakthroughs he said will remain less certain and that, you know, Ji Gen ai, sorry, is gonna make a lot of impact on that. But we sort of moved to that, kind of paradigm for drug development. And he talks about a hybrid biotech going hybrid, and I thought this was interesting, he said, companies will increasingly cherry pick talent and capabilities across regions. And the West absorbs part of the China recipe. So I think that, you know, he's creating this notion of bridges between the U.S., Europe, and China. More bridges, not fewer bridges, and I, those are just a couple, Selina might talk about some of the other disruptions, but the ways that he's, you know, you are right, Europe is a cautionary tale, but he is actually talking about multiple bridges now across the regions.

Selina Koch:

Yeah. Selective bridges, I think is, is really the message. You know, he, he acknowledges there might be some points of strategic leverage where you could justify the US maybe decoupling it a little bit like API manufacturing would need to not be so dependent. But for the most part, you know, he says like, logically it doesn't follow that you then just sever everything by default. Because what you really need is to take the best of all these different regions and marry them to fix an ecosystem, which is, you know, stumbling along. So very slowly right now and, we all know how fast things are moving in China. It might do us all good to think about which pieces of that we could import, right?

Simone Fishburn:

And Selina one follow up to that is, I mean, this is something that I think is at least five, maybe 10 years away, but he talks about even going beyond the 55% reliance on the US market, with Europe, China and Japan capturing more, but he's now also broadening to the Middle East, right? We don't talk a lot about the Middle Eastern countries, which are starting to get very interested in, the sort of drug development or biopharma. ecosystems and, other parts of Southeast Asia. Obviously we have a focus on Korea, but these various other regions are also becoming a little bit more prominent, in the ecosystem. And I think that's extremely interesting in a space to watch.

Steve Usdin:

Just quickly, I'll put in a plug also for our Bio€ quity meeting that's coming up and point out that there are also emerging centers of innovation in central Europe.

Simone Fishburn:

Right. Because our Bio€quity meeting is in, Steve?

Steve Usdin:

Prague.

Simone Fishburn:

And the dates are?

Selina Koch:

May four to six.

Simone Fishburn:

there you go, everybody. This we'll have rehearse that in the future a little bit better, but okay. We did okay.

Jeff Cranmer:

Yeah. So yeah, and our interest goes well beyond Korea. I mean, we haven't, dug as deep into innovation in Southeast Asia. It, it's not beyond Singapore. It's not really moving the needle yet, but obviously a lot going on in Japan, Taiwan as well. Um, stuff we're gonna cover and Bio€quity, Steve, glad you bring it up. It's our 26th Bio€quity Europe. We pick a different city every year. I am currently recruiting innovative early stage private European companies to join the presenting class. And get your tickets now, what more can I say? I, I think the rates are better now, but also this meeting sells out every year.

Simone Fishburn:

Pop quiz, Jeff, when are we gonna be back in Europe this year?

Jeff Cranmer:

Oh, I wanna say September. I'm

Simone Fishburn:

Go ahead. Good month to say.

Jeff Cranmer:

I, I'm feeling, um, a little Grand Rounds. I'm, I'm feeling like I could get my geek on. Where are we holding it this year?

Simone Fishburn:

that will be in the lovely city of Amsterdam.

Jeff Cranmer:

Do they have butteries in Amsterdam?

Simone Fishburn:

Oh no, they do something else over there.

Steve Usdin:

They have some other things in Amsterdam.

Jeff Cranmer:

Yeah, I, I was so fascinated when we, when we, uh, had our last Grand Rounds in, a very esteemed university you may have heard of in, in the U.K. and, uh, we had a, uh, a fierce debate between, Steve Bates and, uh, who did he duke it out with? Yeah, the

Selina Koch:

Yeah. Van Biervliet.

Simone Fishburn:

Jerome? Yeah.

Jeff Cranmer:

And, uh, I think a buttery was involved. But anyway, that kind of sounds like a U.K. answer to the US steel cage match. But, uh, what can I say? We're gonna take a quick break and we're gonna come back and we're gonna talk rare diseases.

Alanna Farro:

BioCentury This Week is brought to you by The 5th East-West Biopharma Summit in South Korea. An arc of innovation is emerging across Asia, and Western biopharma leaders are taking note-from cross-border deals to newcos. In March, 2026, The 5th BioCentury-BayHelix East-West BioPharma Summit visits South Korea for the first time. Meet the biopharma leaders putting Korea innovation on the global map. Learn why Korea has become a clinical trial in manufacturing hub. Discover if Korea is the next hotspot for NewCo formation. Plus, meet biopharma innovators from India to Singapore, to China and Japan. Register now at BioCenturyEastWest.com.

Jeff Cranmer:

All right, we're back. Tierney. Welcome to the podcast.

Tierney Baum:

Thanks for having me excited to be here.

Jeff Cranmer:

Yeah, it's good to get you on your first one and, uh, we look forward to many more visits. You've had a, a lot of, uh, a run of great, analysis in BioCentury lately. And this one is very timely as Saturday was Rare Disease Day, and, uh, you took a look at Stargardt's which is a rare inherited childhood blindness. Why was now the right time to write about this?

Tierney Baum:

So, you know, the impetus for this story was really, new Phase III data that came out at the end of last year from Belite Bio. And it's really the first time we've seen late stage data kind of reaching, reaching its endpoints and their, their position to have to have the first approval in the next year, because of the promising data. So, the way this drug works from Belite is it's an oral small molecule. The more advanced programs in this are really targeting a more substrate reduction strategy, so a lot of the pathology that happens in stargard disease comes from the inability of a protein called ABCA4 to traffic, visual cycle byproducts out of the cell. And so when that protein is dysfunctional, these byproducts accumulate and that leads eventually to, photoreceptor dysfunction degradation, and then the blindness that we see in patients. And so by targeting different proteins that reduce the incoming substrates or incoming retinoids into that cycle. These companies are effectively trying to slow down the visual cycle, and slow down the accumulation of these byproducts that lead to toxicity in the retina. And so these more advanced programs are well positioned for patients who are on the earlier end of the disease spectrum. So because it's a progressive disease. This is a strategy that will really delay disease progression. And so patients who are earlier on have more to gain. And then in writing this story, I realized that this was actually gonna be a big year for Stargardt Disease. There's about six upcoming milestones and many of those are actually in kind of a new wave of therapies. Many of which are gene therapies and, a few that are really trying, some innovative approaches that could even restore vision in more, advanced patients.

Steve Usdin:

I just wanna jump in there because I think that's really extraordinary. If you think about it, if you think because most of the interventions for progressive diseases are aimed at stopping the progression. But to be able to actually reverse it and then to be able to reverse it in such a dramatic way that people will have restoration of vision you know, it's just extraordinary.

Selina Koch:

How much reversal is, is on the table here. So if I've understood you the AAV gene therapies are essentially simple gene replacement. Mm-hmm. And so they could, for the cell, if you treat early, I suppose there are cells there that are, have become less or non-functional, but are not dead yet. So to the extent that you could rescue their function. If you could have an improvement in vision, probably. But you might expect these initial therapies mostly to slow progression still,

Tierney Baum:

Right.

Selina Koch:

I think. Or how do, how do you think about that? I think that's right. And I think still the AAV therapies as many AAV therapies, they do offer a potentially curative option. But again, that's more the damage that's been done has been done. Right. we're not regenerating photoreceptors in any of these circumstances. But I will say for the patients who have still intact photoreceptors, but are just at the more beginning stages of toxicity accumulation, some of these gene therapies we're starting to see that there are actual visual improvements happening not just delaying progression of these patients.

Simone Fishburn:

Tierney I have a question. I mean, there are a lot of, other retinal diseases beyond Stargardt's, right? And if you are taking a strategy, I understand the toxic metabolite is unique to maybe this particular pathology. But if you are taking a strategy that is is about replacing photoreceptor function, wouldn't that be applicable to you know, other inherited or even non inherited eye diseases where, where there is degradation.

Tierney Baum:

Absolutely. Uh, so first of all, I will say I think the substrate reduction strategy is in other places we see this in other degenerative disorders. So it's not totally unique to Stargardt and may have outside applications. But I think in terms of, I think you're right on with the eye diseases. I mean, most of these companies have platform tech. And so this is a really good high interest area where they can validate these platforms and, and it's a great test case where they can then move on to broader inherited retinal dystrophies age-related macular disease. And that is obviously a much broader market.

Selina Koch:

Yeah, it's, it's, so look back in 2017, we probably all remember the approval of Luxturna, right? It kind of paved the way to show like you can treat these inherited eye diseases. And then the eye, while it's a small organ, it's kind of separate from the rest of the body. So you can use very small doses, right? So it's no secret, it's been a tough year for gene therapy, a lot of safety issues. Um, but if you're putting very small doses into this little compartment that's sort of separate, well, you're not gonna kill somebody, right? So in this era where gene therapy is, um, a little out. In terms of investor excitement, this might be a place where it could continue, but my understanding is that there are some practical challenges, right? So everybody wants to do biomarker driven development. But how do you track target engagement and mechanistic changes of your drug in this little compartment? Like you're not going to be measuring blood biomarkers. Um, I don't think you're gonna be biopsying retinal tissue per se. So there's a host of challenges, right? Um, but if you back it up a little bit, I think from the technology perspective, some of these companies are solving some of those challenges. So this gene that needs to be expressed, it's really big, right? So what's being done to address that?

Tierney Baum:

Yeah, I, I think that brings up an interesting, kind of unlock that's happened recently in this area where there was definitely a barrier before for the gene size that a single AAV could deliver. So ABCA4 is 6.8. Kilobases, which is bigger than the five kv that one AAV can handle. So in the last couple few years we've really started using split AAV technology, and so that's been become very attractive for delivery of larger genes like this. The problem though is that. as you would imagine those, if you split up an AAV, they have to find each other in the cell, so it's less efficient than if you were to just do one type of delivery. So there's a few different strategies going on that these companies are adopting to make that more efficient. I would say the most common one is a split-intein strategy. So basically there's these signals attached to each end of, of each AAV, they get, transcribed into mRNA and then translated into two halves of a protein. And then those split-intein in theory are able to find each other and then become reconstituted into a full length protein inside the cell. But I think that, that, again, you know, coming back to the trial that creates, there's going to need to be a lot of benchmarking in terms of, you know, are they really getting full protein expression, are they looking at the biomarkers that are showing that the AAV is getting in there and finding each other?

Selina Koch:

So you, you mentioned the most advanced ones being small molecules. This next wave is genetic medicines. When are we gonna start to see data from them?

Tierney Baum:

Yeah. So I think almost all of the upcoming gene therapies are replacing the ABCA4 gene, and those are all fairly early stage still, Phase I or Phase I/II. I think a couple of those we will see data by the end of this year. Interestingly, the most advanced program is actually not in that gene. It's in a different gene called RAR alpha or RAR-α, and that one's in Phase II and it has preliminary data. And, it has shown, RAR-α is like an parallel and upstream regulator of various homeostatic pathways for photoreceptors. So it's kind of an indirect upstream regulator of ABCA4, and so this preliminary data has actually shown, really promising early results that it can slow the growth of atrophic lesions in patients, I think they saw a 54% rate of reduction in growth at 12 months. And that's both faster and more of a reduction than the current Phase III data we're seeing from the substrate reduction strategies. So while that's still very early, I think there's a lot of promise that, that target might have more efficacy.

Selina Koch:

Cool, and just from a neat technology perspective, I noticed this pipeline does have an optogenetics molecule in it. When I was in grad school, we thought of that as a research tool. So you could express a light sensitive pigment in a cell of, of choice in the retina and shine a light into the eye and you could induce activity in a specific circuit, right? So, it's cool to see it moving in into the, into the industry as possibly a therapeutic approach. I dunno, if you wanna give a very brief.

Tierney Baum:

Yeah. So I mean, I think, I think the, there are a couple programs that are, doing, like I said, really innovative things with optogenetic type approaches. And so they're actually not targeting the retina at all. And a lot of these programs might even be best for more late stage severe patients who have most of their photoreceptors degraded. But for example, Nanoscope has an optogenetic therapy called MCO-010. So basically there's, you know, a couple intermediate cells between the retina and the brain, right? You've got bipolar retinal cells, and then you've got the retinal ganglion cells. And so those cells help pass signals when the light hits the eye, help pass signals from the retina to the brain. And so, what Nanoscope is doing is creating, an AAV deliverable light engineered Opsin. that allows the bypassing of photoreceptors altogether, and then bipolar retinal cells become activated, passing the signal onto the brain. And restore vision, like I said, this might be a therapy that's a really cool option for patients that are, have lost most of their photoreceptor function.

Selina Koch:

And there's no reason why they can't. I mean, in theory you couldn't use multiple approaches, for people.

Tierney Baum:

right? Mm-hmm.

Jeff Cranmer:

From, uh, baseball and tennis to geeking out about, uh, cool stuff in biotech. That's what we deliver here on the BioCentury This Week podcast. Steve, there were some interesting comments made. Thursday on CNBC's morning program, by FDA, Commissioner Marty Makary, why don't you fill u s in.

Steve Usdin:

So really look, his comments, were extraordinary for anyone who has experience in the life sciences who's been around for a while, I think maybe some of the people who are watching and listening who are newer to it may not have realized, how extraordinary the things that he said are coming from an FDA Commissioner. First, he talked about a pending regulatory application. That's something that FDA officials for very good reasons, never did in the past. He seemed to be talking about uniQure's Huntington Disease Therapy. He denigrated it in a very crude way. The company shares sank 30%. and I think that the hopes and expectations of Huntington's disease patients went down far more than that. Second, he made it clear that FDA leadership has become politicized. He said that CBER Director of Vinay Prasad once supported Bernie Sanders, but now he's an ardent supporter of Donald Trump, no FDA employee should ever be described in terms of their political affiliations. What kind of message does that send to the staff? To drug developers, to patients? You know, it, it's just something, again, that's extraordinary. Third, he described Prasad in a very interesting way. He said, he's "on loan" from UCSF, that's really odd. Previous center directors were career staff who were there for very, very long periods of time. They weren't on loan. Marty Makary, came from Hopkins to be the FDA Commissioner. Is he on loan from Hopkins? I don't think so. Another thing Makary he said a couple of media outlets have issued fatwa's against Prasad, and he used that term fatwa twice. I guess he's talking about me along with some others. I wrote the first editorial that I saw anyway, calling for Prasad to be, pushed out. Makary should know what a fatwa is, and a fatwa isn't journalists writing stories that he doesn't like, you know? All of that I found extraordinary.

Simone Fishburn:

Steve, I mean, I wanna just call, call it like it is for some of these things. Right. So Prasad is on loan, quote unquote. Well, my understanding is he still lives in the Bay Area and he comes out x number of times per month. Right.

Steve Usdin:

He's, he is about three times a month.

Simone Fishburn:

Right. Okay. But he lives there rather than here. We've actually seen that even in the corporate setting where it didn't, for GSK for example, it didn't really work out. It's very difficult to do this remotely. I think it's fine for the FDA to say, we don't have to be so DC centric. Let's have offices all over the country, but that's not actually what's happening. It's more like I'm only putting one foot in is the message, right? And so it seems to be a defense of that. And the other thing is you obviously, as you pointed out, did call for that, but there's been a lot of press in other major journals, including the Wall Street Journal, which is read obviously by a lot of Republican readers and business folk as well, that's been very, very critical of Prasad. And I think it's really hard to to hear the statements from Makary without hearing into them defensive of Prasad. Like, you know, it's as if there's a momentum towards his getting, maybe the end of his tenure. It's never good when the White House has to step in and influence policy as they did with the reversal on the, Moderna flu vaccine. And all of this is against a backdrop that I'm sure all of our readers are reading about in the broader press regarding MAGA and MAHA taking a different tone on vaccines as the elections this year near as the midterms near on the grounds that people aren't happy with the anti-vaccine stance

Steve Usdin:

Yeah, so, so look, you know, yeah. So, so I'd say, you know, look it, this all plays into something else too. It, it really isn't clear, how much longer Prasad's going to be at FDA. I had thought that he was gonna hang on until the midterms. I don't think that anymore. He was absent for the public rollout of the plausible mechanism pathway. Which McCarey said that Prasad wrote. I've heard other reporters have confirmed that Prasad is being investigated for harassment, both sexual harassment and more generally for unprofessional treatment of subordinates and colleagues. Of course, he should be presumed innocent until an investigation is completed, but there does feel like there's a mounting pressure for him to go. The real question I think will be if he does or when he does, whether he'll be alone, or whether Makary would follow afterwards. I still think that M stay in place until, the midterms, that's the most likely.

Simone Fishburn:

I wondered about that, Steve, is, is, you know, given what's gone on, is it possible that he could be the sort of, I don't even know if I wanna call it sacrificial lamb, certainly a lot of the things he's triggered himself, but, is that a way to sort of blunt this issue, removing him, you think?

Steve Usdin:

I, I think that there's, look, there's a, there's a, a tremendous amount of frustration and anxiety about what he's done and what he is doing within FDA, in industry, and politically. And interestingly it comes from both sides on some of these issues. So, for example, on the mRNA vaccines, you know, his initial decision to refuse to review Moderna's vaccine. It was highly controversial and it raised a, a real firestorm, and FDA had to walk that back. On the other hand, the leaders of the MAHA movement have said that if FDA approves an mRNA vaccine, that MAHA voters will stay home, for the midterms that they won't support Republicans. That creates a real, kind of dilemma. If you have an FDA that's making us decisions based on the directions that the political winds are blowing, rather than taking a step back and saying they're really looking solely at what's best for the public health.

Jeff Cranmer:

So Steve, uh, the Plausible Mechanism, you said it was rolling out, what's going on with it?

Steve Usdin:

So, FDA has released draft guidance now they're calling it a Plausible Mechanism Framework, rather than pathway. And I think, generally it's, it's gotten, you know, really, good reviews. People think it's a solid step forward. It's also a step forward that they're doing it in this way. They're releasing it as draft guidance. There's a docket open patient advocates, academics, companies can comment on it and they can see something in black and white that gives them an idea of, of what it is. And I think as, as far as it goes. As I said, you know, generally people think it's, it's going in the, in a, a good direction for rare disease patients. There are some people who think that it could and should go further. It seems to be primarily oriented toward, people say N of one, but it's really n of a handful because usually when there's one they, there's another somewhere. But it, it seems to be oriented primarily to bespoke therapies for extraordinarily rare conditions. And that's what was discussed a lot at the, rollout press conference. I think that there's a hope in some quarters that they can find ways to move the approaches that are described in the draft guidance little bit more broadly, while still keeping, you know, guardrails around it to make sure that, the therapies that should be developed through, more traditional um, evidence generation pathways are. But that the, these kind of pathways that are described in the draft guidance are available beyond simply n of one therapies. Tracy Beth Høeg, was at the, uh, rollout and she said that, um, she has an expectation that they will be used more broadly.

Jeff Cranmer:

Alright, well thanks for that, Steve. you can find, many of the things we talked about on BioCentury.com, including, Tierney Stargardt piece, also on BioCentury.com Korean biotech is punching above its weight in neurology. Selina and our colleague Lindsay Martin took a look at the growing unpartnered CNS inventory that is meeting rising Western appetite for BBB delivery and next gen biologics. And Stephen Hansen, our man on the obesity beat, took a look at a few readouts from Novo. One that, uh, shave billions in market cap, off the company, that's the CagriSema data of course, but also, uh, some more positive data out of the company. So head to BioCentury.com to check that out. And, uh, Franck Le Deu's commentary, that's in front of the paywall. So, uh, hit up the link in the show notes. And while you're there, Why don't you give us a rating, leave us a comment, drop us a question. Always look forward to hearing from our listeners, and we'll be back next week. Stephen, will be hosting as Simone and I will be off in Korea, on stage at our conference. Uh, that's the East-West Conference. All right. Thanks for tuning in. Kendall Square Orchestra provides the music for BioCentury's this week. The group connects science and technology professionals and other members of the Greater Boston community to collaborate, innovate, and inspire through music, while supporting causes related to healthcare and education.

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