BioCentury This Week
BioCentury's streaming commentary on biotech industry trends, plus interviews with KOLs.
For three decades, BioCentury has helped biopharma executives and investors make business-critical decisions and build larger networks with peers across the innovation ecosystem.
BioCentury This Week
Ep. 364 - UCB’s Candid buy, Grand Rounds and leukodystrophies
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UCB is the latest biopharma player to consummate a multibillion-dollar deal amid a surge in biotech M&A activity, acquiring Ken Song’s Candid Therapeutics, one of the leading T cell engager companies. On the latest BioCentury This Week podcast, BioCentury’s analysts discuss the $2 billion takeout and the opportunity in the TCE space.
They also assess the drug development landscape for leukodystrophies and the market for biotech IPOs on NASDAQ, where a trio of biotechs exceeded expectations last week, lifting hopes that the window for new listings is broadening. Looking ahead to June, the analysts also preview the third BioCentury Grand Rounds U.S. conference. This episode of the BioCentury This Week podcast was brought to you by Jeito Capital.
View full story: https://www.biocentury.com/article/659365
#BiotechMA #TCellEngagers #Leukodystrophy #BiotechIPO #TranslationalScience
00:01 - Sponsor Message: Jeito Capital
02:19 - Grand Rounds Seattle
12:47 - UCB’s Candid Buy
20:27 - Leukodystrophies Pipeline
27:22 - Biotech IPOs
To submit a question to BioCentury’s editors, email the BioCentury This Week team at podcasts@biocentury.com.
[Autogenerated Transcript]
Voice Talent:BioCentury This Week is brought to you by Jeito Capital, a leading global independent private equity fund with a patient-benefit driven approach, aimed at financing, and accelerating the development of ground-breaking medical innovation. Jeito’s unique investment strategy combines significant capital and collective expertise to support biopharma companies and their management teams-ranging from clinical development to market access for cutting-edge innovations—with one main objective: go faster for the patient. Learn more at jeito.life
Jeff Cranmer:Another week, another multi-billion dollar biotech takeout. This time the deal is in the hot T cell engager space with UCB. Taking out Ken Song's Candid Therapeutics to the tune of $2 billion upfront. We'll discuss the deal on the latest BioCentury this week podcast. We'll also talk about leukodystrophies, an area where drug development remains limited, but we are starting to see some momentum and turning to the public markets, three biotechs raised more than 850 million in upsized IPOs, and then they gained in the aftermarket. Always good news. I'm Jeff Cranmer host of the BioCentury This Week podcast and joining me to discuss all of this are my colleagues.
Selina Koch:Selina Koch, Executive Editor.
Lauren Martz:Mars, executive Director of Bio. Pharma intelligence.
Danielle Golovin:Danielle Govin, Senior Biopharma Analyst.
Jeff Cranmer:All right. Thanks for joining me. Uh, May 4th, which means our Bio€quity Europe conference has kicked off in Prague with a lot of great panels and presenting companies up on stage. I think they even did the dreaded fun run this morning. Um, fun and run. They do rhyme. That's about it. However the podcast host sits, so good on everybody that uh got out there and uh hopefully you had fun. Well, with Bio€quity kicking off, that means one thing for us here, those of us that didn't get to go to Prague, we are hard at work getting ready for BioCentury's third Grand Rounds. it'll be in Seattle next month. And here to tell us about it is my colleague and neighbor Karen Tkach Tuzman. Karen, how you doing?
Karen Tkach Tuzman:Thanks, Jeff. I'm doing well. Uh, it's all grand rounds all the time for me, and uh it's our third U.S. meeting, but it's actually our fourth one overall because we've taken it to the European U.K. area last year with Cambridge U.K. and this year again with Amsterdam uh in September. But first we're going to Seattle in June, which I am very excited about. first of all, Seattle in the summer is beautiful. but also it is a major epicenter of protein design revolution among other innovations. And we're just really excited to highlight the local ecosystem and bring our folks out there to talk some deep, fun science.
Selina Koch:Give us a little preview of, of the enabling technologies that might be featured there. That's a very like sciencey technologically geeky area of the conference that I always love and look forward to.
Karen Tkach Tuzman:That warms my heart because I too love the enabling technologies, extra geeky corners of the conference. So what Selina's alluding to is that um the BioCentury Grand Round series really gets at translational science across a really broad important spectrum of highlighting emerging disease biology. So target biomarker discoveries, what we're understanding about certain disease areas or pathways the enabling technologies fronts, she mentioned uh which are the tools that transform how science has done. And then we also have. an area track around therapeutic modality design and getting the geeky details there. With an enabling technologies, we will be getting into a couple things. One uh your, everyone your favorite in mine, everyone, the tool on everyone's lips uh transformer architectures. so these are the model structures that underlie the LLMs that have come to. Occupy so much uh activity in the world these days, and you keep hearing about people throwing that at biology, but what does that actually mean? That's what we'll be getting into in that panel. There's also what I wanna highlight we call it a horizon session because we'll be having a brief keynote followed by a panel, and this will be focused on a local thought leader, Jay Shendure. He's got many, many affiliations uh University of Washington, HHMI, et cetera. and he's the lead scientific director of the Seattle Hub for Synthetic Biology, where they're exploring the DNA typewriters technologies lab developed. This is a really interesting technology that basically uses DNA, and CRISPR based modifications to record a cell's lived experience. So what's happening inside a cell? What did the cell see? and that allows you to one, potentially better understand what's happening in the evolution of disease through disease modeling. if you can actually peer, get a record of what the cells saw while those processes were happening. But the other side of it is for programmable biology. It's the sensing part of a sense and response. Uh, so what could you do to cell therapies, for example, with a DNA typewriter in the mix? These are some of the geeky questions we'll be getting into on the enabling technologies front.
Selina Koch:And gates or gates, so exciting.
Jeff Cranmer:should be fun. Who? Uh, David Baker will be there. Yeah.
Karen Tkach Tuzman:Of course, yes. He uh kicks the whole thing off with a keynote on June 3rd diving in, you know, we all know about uh his great work with protein design, with protein structure prediction. and this is kind of talking about the next generations and threads of that work around catalysis around intrinsically disordered regions of proteins, those kind of harder problems. Next rungs for his lab's underlying technology. and of course there's a whole uh robust startup ecosystem there with technology spun out of his labs um and others. where we uh are really looking to highlight the great stuff going on.
Selina Koch:Well, not to um only single out the enabling technologies here. Let's hear maybe just a little bit about something we're gonna be featuring on the new biology front and then on the therapeutic modality front.
Karen Tkach Tuzman:On the new biology front I'm definitely excited about the panel on preeclampsia. So this is a pregnancy complication that affects 8% of pregnancies worldwide. it's one of those classic, like very large women's health markets, very underdressed. But as Danielle has captured there's been some progress on the clinical front with results from DiaMedica who will be on the panel. And also we keep seeing in our literature monitoring more and more sort of papers pointing out better understanding of the disease biology signals in a biomarker or potentially, you know, diagnostic way. There's a major diagnostic that is established in this space, so we'll be looking at that and going beyond that. that's an area I'm really excited to talk about, sort of the underlying biology of preeclampsia and, and the directions forward. and then on the modalities front, you know uh it is Seattle uh home of Pfizer, but before that Seagen. so we, the ADCs legacy there runs deep. Um, and we chose to focus in, in classic Grand Rounds style on a sort of, Nerdy and underlooked, but important part of the ADC product, which is the linker, the linker chemistry. And when you dig into that, you realize,'cause you think, oh, a linker, you kind of think of it as just like one little piece connecting this to that. But actually there's so many dimensions. To the linker science, you know, on the antibody grabbing end, on the payload grabbing end on the various ways, you know, is it stable, is it unstable? And actually is unstable better than stable sometimes there's a lot of niche issues uh there that, well, they sound niche, but they're actually kind of the make or break for an ADC. and so we'll be digging into that with, People who really focus in on that area. I'm excited about that one.
Selina Koch:Before we get to Grand Rounds, we will publish on this topic looking specifically at progress being made in site specific conjugation techniques. So we have a slide deck underway for that.
Karen Tkach Tuzman:Yeah. we, I really appreciate Grand Rounds because we nerd out on specific scientific topics that are we, when we pick these topics, we select for areas where there's momentum and bottlenecks, we try to get at what is the scientific directions that could overcome those bottlenecks to break through to fully realize the momentum. And then we also have uh some important strategy sessions where, you know, we get higher level perspectives. For example uh Simone will be sitting down with Takeda, CSO, Chris Arendt and they'll be looking at um I think a topic on a lot of people's minds is what does it actually mean for ai you know, rubber to hit the road for R&D. we will have some important buy-side views kind of giving uh particularly the innovators at that academia industry interface, what they really need to think about if they're trying to commercialize their science in 2026. and we've got some really interesting workshops around indication selection, like how you get that right when you know you've got your, you've built your cool hammer and, but you gotta pick your nails. And an expanded view of a target product profile, like what does that mean in 2026 to really do that, to the maximal capacity to advance your technology. So these are the kinds of things we dig into and it's also just fun. It's a fun meeting where people really let the nerd hang out and I am so excited to welcome you all there.
Jeff Cranmer:Yeah, and we'll have some investors there of course uh that's a big component of this meeting. Our founding investor Xontogeny that's Chris Garabedian shop, affiliated with Perceptive and uh another component presenting companies. I am uh actively recruiting, presenting companies. And Karen is a big part of that as well as we get some really early stage that is way above my pay grade. So guess when Karen comes in and is just like, oh yeah, this is the new hotness. And indeed we had, uh, one of our podcast listeners reach out to us to express interest in presenting at the conference. I was delighted by this the company is AddGraft. It's a University of Chicago spin out. It's raising a pre-seed round, so right in the the wheelhouse of Grand Rounds, and it is developing an autologous engineered skin cell therapy platform for a rare disease. Shout out to the CEO who uh raised his hand and uh will be on stage next June. And speaking of getting our listeners of the BioCentury This Week podcast to Grand Rounds well, our advanced rate expired on Friday, but hey. for our podcast listeners we are gonna offer an extended advanced rate for delegates and presenting companies, so you can reach out to conferences@biocentury.com to get a special code to sign up. Look forward to seeing you all, at Grand Rounds, and of course Selina never lets me slide unless I talk a little sports uh for baseball fans the Mariners are in town. They'll be playing the Mets on June 3rd and June 2nd and 1st. And so, hey, I know it's a short hop from Vancouver to come down short hop from California, but hey, if you're in New York City and you've got some hot new technology or wanna check out the hot new technology and you also happen to be a fan of the beleaguered New York Mets, come on out to Seattle, get a little biotech with your baseball. Okay. thank you for that KTT. I know you're a busy woman. Appreciate you coming on. Let's turn to UCB. Now, UCB is building its immunology pipeline with its $2 billion acquisition of leading T cell engager company Candid Therapeutics. Candid launched in fall of 2024 via the NewCo model. That's something we've talked a lot about here, where Western players fund a new company uh around assets sourced in Asia, most typically China. Candid was backed by a syndicate of roughly 20 companies. The company was poised to go public via a reverse merger with Rallybio, with a big syndicate set to provide 505 million in fresh funding. Rally will take its termination fee on this one. And Ken Song, it's the second company sale in as many years for the CEO of Candid. He was CEO of radio pharma company, RayzeBio, which went public three years into its lifespan. And then in January, 2024 was bought by Bristol Myers for 4 billion uh 4.1 billion to be precise. Uh, Lauren has been following the TCE space very closely for us here at BioCentury. Lauren, what did UCB see in candid to drive this deal, given the size of the TCE pipeline, that's uh that's out there?
Lauren Martz:Yes, thanks Jeff. so I think what they likely saw was a little bit of clinical proof of concept. The pipeline of T cell engagers for autoimmune diseases, as you mentioned. Is large and growing. And we don't have a ton of clinical data proving that this concept that there is other evidence to support actually works in autoimmune diseases and you know, the best way to do it in autoimmune diseases. So Candid is one of the few companies that has reported some data um some Phase I data specifically for their lead, BCMA, CD-3 T cell engager. I mentioned there's support. We've talked about this before. We know that depleting B cells. With a T cell based mechanism, CAR T specifically can reset the immune system in autoimmune diseases. There is plenty of reason to believe that T cell engagers can do something similar because we've seen them do something similar in cancer. and I think the question that we've been thinking about, and we have a recent slide deck that gets into this, is again, how to do that the best way. If we can take all of the T cell engagers that have been tested in cancer and put them into autoimmune diseases, or if we have to um develop something specifically for that. So, Candids lead programs were originally developed for cancer and moved into this application. They have some earlier stage trispecific T cell engagers that are specific for autoimmunity. And I think as more evidence comes out from this program and from the others that are in Phase I, I think we'll start to get some more information on that.
Selina Koch:From the UCB side of this deal, this one is an acquisition, but I think it did a licensing deal not that long ago for another T cell engager which I believe has a different target. And as you pointed out in your big analysis recently. There's like three main targets, right? Um, so are we gonna see more of this, like the larger companies trying to like, cover the bases of those three targets?
Lauren Martz:I think we will. I think, you know, that's exactly what we've seen from UCB here. So Candid programs, its lead program targets BCMA. It has another one against CD-20, and then some trispecifics that hit multiple targets. What UCB got from a company called Antengene in March is a CD-19, CD-3 T cell engager. Again, we don't have a ton of data to support where exactly each one of these targets will be most useful within the autoimmune disease landscape. But there are certainly hypotheses about where you could apply each of these. So depending on the severity of the disease, the type of B cell that that is specifically involved and different targets will, will have different applications. When you look at the pipeline of companies that are working in this space, especially thinking about the pharmas and the potential buyers of companies like Candid, there do seem to be some holes, you know, maybe different pharmas don't necessarily have all three of these base covered at this time. And some of the programs that the pharmas have in development are things that they've repurposed from cancer. Um, you know, some of the earliest data that we have is from Amgen's Blincyto, for example, and that is, you know, very solidly a cancer therapy and the market potential of that one, as you think about autoimmune diseases is not clear just given its um its delivery mechanism. I think there are certainly more opportunities for all of these biotechs that are starting out in the clinic with T cell engagers for autoimmune diseases to have partnering opportunities.
Selina Koch:Right, and I think the comp that kind of comes up most frequently in this conversation is Cullinan, which you also include in your latest set of stories. Presumably it was considered, you know, in, in this process um that wasn't the one that was acted on it at this time, but perhaps in the future.
Lauren Martz:Yes, I think Cullinan was the first biotech to start a Phase I trial with a T cell engager in autoimmune diseases. And they do have, I think, a bit more data than some of the other companies. So you know, they may have been considered, they may be an acquisition, acquisition, target themselves. I guess we'll see.
Jeff Cranmer:Yeah, investors think so. I mean the stock popped up 18% out of the gate this morning. holding steady as we record this around 9% company's market cap just under a billion. We'll see what happens. All right, and uh as I said at the top there was gonna be a pipe and well there was a whole bunch of investors backing this deal. They won't get their payout now. And those investors in Candid whether or not they jumped in on the pipe, they're gonna get their payday a little bit sooner. All right, we're going to go to a quick break and we're gonna come back and we'll talk Leukodystrophies. Um, we had a great deck from our colleague Danielle Golovin.
Voice Talent:This episode of BioCentury This Week is brought to you by the 3rd BioCentury Grand Rounds in Seattle. Advancing drug development requires more than discovery. It requires the right partners. The 3rd edition of BioCentury Grand Rounds U.S. convenes venture capital, biopharma decision-makers, and academic innovators in Seattle, June 3rd to 5th. This R&D-focused forum brings together leaders at the forefront of translational science to examine the breakthroughs, bottlenecks and strategies shaping the future of drug and diagnostic development and how to make early-stage R&D investible. Discover cutting-edge disease, biology, and platform technologies. Gain insights from emerging biotechs and academic pioneers. Schedule partnering meetings with VCs, pharma, and leading academics who can accelerate your path forward. Grand Rounds U.S. is where rigorous science meets strategic capital and where the right conversations move discovery toward development. Join us in Seattle and discover what's next in biopharma and who's driving it. Secure your spot. Register at BioCenturyGrandRounds.com.
Jeff Cranmer:We are back. Drug development remains limited for leukodystrophies but more than two dozen biotechs are now matching therapeutic modalities to the genes, pathways and cell types driving distinct forms of white matter disease. Danielle, you took a deep dive here, created a great deck with all sorts of uh pipeline charts and uh cool graphics. Why now to write about Leukodystrophies Danielle?
Danielle Golovin:so Leukodystrophies, just to give you an overview or a group of rare genetic and progressive disorders that destroy the white matter of the brain and spinal cord. That's the myelin sheath that insulates axons and allows the different brain regions to communicate as functional networks. So you can imagine how disrupting that network is devastating and leads to cognitive and motor impairments. What signaled me to write this deck was that in March FDA, accepted and granted priority review to an NDA for Zilganersen. So this is an ASO from Ionis, with a PDUFA date in September. So that's for Alexander Disease. And when I looked that up in BCIQ, like, you know what is Alexander Disease? Turns out it's a rare leukodystrophy that has no other products in the pipeline. So that made me kind of zoom out. And what are leukodystrophies and what other pipeline activity are we seeing?
Selina Koch:So among those 50
Danielle Golovin:Yeah. It turns out there's 50. Who would've?
Selina Koch:Collectively there's a lot of leukodystrophies, but like my impression is not that many of them have drug drug development activity. Which would you find there?
Danielle Golovin:Oh yeah, that's true. So we found about 20% have at least one program. Not that there's high activity in 20%, but there is some activity. And so we found 11, I mean, there's no exact number. It was around 50, right? That leukodystrophies, but yeah, 11 subtypes we identified that have a pipeline and out of those, there's only an approved drug in three of them.
Selina Koch:Right, so an area of uh high unmet need lots room and where there is activity, it seemed like it was mostly gene therapies and small molecules. So this Ionis news, sort of like ushers in another kind of treatment strategy. Is that how you think about it?
Danielle Golovin:Yeah, exactly. The ones that are approved, we have two gene therapies and two small molecules across three indications because one indication has two small molecules approved. The overall pipeline in the 11 subtypes reflects that, right. We, we mostly see small molecules and gene therapies. It's actually pretty split, evenly. But then, yeah, this, this RNA like ushers in a new type. And it also has to do with the biology of the diseases, of course. So most of the 11 are a mutation in an enzyme. And so the gene therapy is, you know, replacing that enzyme. We see a couple ERTs enzyme replacement therapies, but then in a smaller subset, of the subset, a smaller subset of the subtypes that we identify that have drug activity, the problem is too much of a protein and that it's like a toxic buildup. And that's where the RNA knockdown modalities come in.
Selina Koch:Right. So when I think of like ERTs and rare disease, it's the classic modality, right? All going all the way back to Henry Termeer and, and Genzyme and all of that. And so, you know, in recent years it's been like another one comes up for like, oh, another ERT for another rare disease. But the ones that you pointed out to here are actually like pretty innovative, right? So tell us a little bit about those.
Danielle Golovin:Yeah, I was actually surprised, to see the lack of ERT in the leukodystrophy pipeline. But then I realized, well, this is a CNS, these are CNS disorders, so probably harder to get that enzyme into the brain. And so the two that we see here have a brain shuttle to improve CNS exposure. One's from a collaboration between Chiesi and Key2Brain and another between JCR and Medipal.
Selina Koch:But even in the gene therapy space, there are effectively ERTs, right?
Danielle Golovin:Yeah, so BioStrategies has like an interesting, I guess it's kind of a combination of ERT and gene therapy because they're encoding the enzyme and also encoding a brain shuttle with it and packaging that in an AAV. So I thought that was one of the more innovative things we saw in the pipeline.
Selina Koch:I mean, the whole concept of vectorized biologics are really interesting, right? Because you. We have this possibility for, for one and done or infrequent treatment, but you also don't get like the peaks and the troughs in, in drug blood levels and things that you would get with periodic dosing. But then we, it also that, you know, so you think there would be a lot of upside there and motivation on the other hand, it, it clashes with, this is just really hard time the industry's been having with gene therapy. So it's interesting to see, you know, still in select spaces, some people willing to make these bets. when we're looking at these more technologically innovative approaches for getting enzymes or genes in into the brain, you know, where are these brain shuttled programs? Are they for specific indication? Are they across indications?
Danielle Golovin:Yeah, so the two that have the BBB shuttled ERTs, are actually in the indications with the highest activity. And when I say high activity, that's still not, super high. The two highest have eight programs each, and that's CRAB disease and GM2 gangliosidosis. Those two are still preclinical, the Brain shuttle ERTs no data on those yet. But in CRAB disease, the most advanced is a gene therapy in Phase I/II from Ajinomoto and Forge. And also GEMMABio and Rare Therapeutics have a AAV gene therapy. They're both AAVs, but different capsids. So we're also seeing that um that'll be a differentiator is like the administration type and the capsid type. We'll see, you know, how that affects the distribution in the brain. And then for GM2 gangliosidosis, we have a small molecule in Phase III from Azafaros. As the leader in that indication.
Selina Koch:Maybe just worth noting for brain shuttle uh fans out there, there was uh FDA, granted its first approval for brain shuttle enabled biologic therapy this year with Denali's MPS II treatment, getting approval.
Jeff Cranmer:Excellent. Well, thanks for that Danielle. Danielle's piece. I'll drop a link in the show notes. Definitely uh shines a spotlight on a uh area where we certainly hope to see more drug development uh emerging, and uh look forward to watching the progress of those already making their way in the clinic or toward the clinic. All right. Turning to the IPO market for biotechs uh IPOs on NASDAQ by a trio of biotechs last week, exceeded expectations. Let's see, who do we have the new kids on the block. Hemab is a Danish company developing a bispecific for bleeding disorders. Company came out of Novo, so Novo Seeds, Novo Holdings backed the company. And uh HealthCap and RA Capital were among the early investors. Uh, Seaport, fans of BioCentury's podcast will remember Steven Paul on with Selina not too long ago talking about his last company Karuna, he co-founded uh Seaport along with Daphne Zohar who also was founder of Karuna. Karuna of course acquired for 14 billion. Seaport is working in the neuro psychiatry space. And fibrosis therapy developer a Avalyn Pharma had a really nice pop. when it got out last week. Selina any thoughts on the state of the biotech IPO market right now?
Selina Koch:Well, it's certainly, I'm, I'm sure lifting hopes that the window could open up a little bit wider for more listings to see three of them do quite well. Get the listing done, but also, you know, do well in the aftermarket. Maybe there's some signal in here though that they're still looking for relatively low risk propositions. You know, I think as, Steven Paul and Daphne Zohar had done before with Karuna, this is, you know, a mechanism that has some validation behind it, but needs a, a solution, a technological to deliver it better and so we're seeing that in some of these companies, that sort of risk profile. But really great to see 3 of them in one week.
Jeff Cranmer:Yeah, Hemab of course, yeah. I, I'm surprised this one just felt like it came off me. I remember meeting with a couple of the Novo Seeds people at JP Morgan, and they were you know, teasing what this company was up to, obviously now in Phase I/II for a couple of different blood disorder indications. I mean, aftermarkets uh where it's at, you know, you get out and then you wanna trade up. And uh earlier this year uh some of the aftermarket performances by newly listed biotechs were a bit tepid, granted, geopolitics may have had something to do with that. But as our colleague Paul Bonanos in his weekly finance report pointed out the VIX has dipped below 20 in recent weeks. That could be a factor here as Selina's pointing out. We also, we saw a crossover round this, this morning as well, Windward Bio, that's another of the NewCos, Windward, $165 million crossover round Orbimed, RA, Janus. A couple of the backers there in this immunology company. That company is entering Phase III programs in asthma and COPD. So again, to Selina's point bit of validation already on the books. Nice to see the crossover round. Maybe that spells an IPO is near. We will keep an eye on what is happening in the IPO market, along with the TCE market. Uh, who knows if we'll see another takeout soon, all eyes on Cullinan. Thanks for tuning in to BioCentury This Week, and a thank you to Kendall Square Orchestra who creates the music for BioCentury's podcasts.
May 15th, that's Friday, May 15th at 7:30 PM at Sanders Theater in Cambridge, Mass. Kendall Square Orchestra performing Brahms Symphony number four and Tchaikovsky violin concerto. so appreciative as always. To Kendall Square Orchestra and appreciative to you, our listeners thanks for tuning in, subscribe to our podcast, drop us a question, and if you are interested in BioCentury's Grand Rounds now's the time to sign up, that is coming to you early next June, if you're on your way back to China from ASCO what better thing to do than to stop off in Seattle and say hello to the BioCentury team and check out these great panels that my colleague KTT told you about? Alright, we will catch you next week.
Voice Talent:BioCentury would like to thank Jeito Capital for its continued support of our BioCentury This Week podcast and our 26th annual Bio€quity Europe conference this May in Prague, Czech Republic.
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