Latest / The BioHub - by Avetix / Episode 102 - Miquel Vila-Perello - CEO of SpliceBio
Transcript
- 0:04So welcome back to another episode of the Bio Podcast.
- 0:07Today we're joined live at Jeffries by Mckelvey of Splice
- 0:11Bio, fresh off the back of getting Series B of the year on
- 0:16Monday at the LA Awards. Michael.
- 0:19Fantastic to have you. Welcome to London.
- 0:21Sorry about the weather. Nothing you could.
- 0:23Do about me, Yeah. How have you found things so
- 0:27far? Great.
- 0:28So thank you very much for having in the podcast a pleasure
- 0:30to be here and tell you about Splice bio and our worked on on
- 0:34Stargard disease. So yeah, yeah, I'm so far great
- 0:36conference. It was great to receive the
- 0:38award kind of the recognition earlier this week.
- 0:42So very happy to to be here. Yeah, nice.
- 0:44It's great to have you in the office for sure.
- 0:46Yeah, awesome. I guess we always get started
- 0:49off with just a little bit of background on who you are,
- 0:52right, and your story and where you've come from.
- 0:55It's a pretty interesting journey, right?
- 0:57So. Yeah, yeah.
- 0:58So I, yeah, I'm sort of a basic researcher by training.
- 1:03I'm chemist slash biochemist. And I always thought I would be,
- 1:08you know, my career would be in academia, basic science.
- 1:11But at some point I really got interested in in bringing the
- 1:13innovations who were developing and, you know, make them a
- 1:17reality for for patients. So I, but my kids in Barcelona
- 1:21then moved to the States. I lived over there for about 10
- 1:23years. I was working with Professor Tom
- 1:25Muir, who has been pioneering the field of protein splicing at
- 1:29Rockefeller and then Princeton. And that's where we developed
- 1:33our core technology. And together with Tom Muir, a
- 1:37former postdoc from his lab, Sylvia Frutos, and actually a
- 1:39friend, you know, a childhood friend from myself who was doing
- 1:43an MEA at Wharton. We founded the company and here
- 1:47we are. I didn't realize we're Sylvia at
- 1:49Princeton as well. Yes, well, she had been at
- 1:51Rockefeller. OK, She never moved to to
- 1:54Princeton. Yeah, but obviously she was very
- 1:56familiar with ideology and it made signs too nice to have her
- 2:00as part of the team. Awesome.
- 2:02And then you, you brought up back to to Barcelona and got it,
- 2:06got it going, Yeah. Yeah, indeed.
- 2:08Nice. Awesome.
- 2:09That's cool. And walk us through a little
- 2:11bit. So obviously ophthalmology gene
- 2:14therapy pretty Goodyear for for that generally speaking.
- 2:18And you obviously mentioned Stargard disease there.
- 2:22And yeah, what what's your opinion on the sort of current
- 2:25state of the, the space I guess in, in that disease?
- 2:28And what are you guys looking to solve with the with the platform
- 2:31you're developing? Yeah.
- 2:33So Stargard disease, as you know, it's a, it's a the most
- 2:36prevalent inherited retinal dystrophy.
- 2:38So it's a disease of the retina where patients basically
- 2:43progressively lose vision. It typically starts with the
- 2:46first second decade of life and then people progressively lose
- 2:50sight. There's currently none, no
- 2:53approved treatments, but it's a disease that affects a lot of
- 2:56people. So there's quite a bit of
- 2:57activity on the space. I think gene therapy approaches.
- 3:01The idea is that, you know, we'll be able to address the
- 3:04root cause of the disease. This is caused by mutations on
- 3:07these very large gene called ABCA 4 and, and basically our
- 3:11technology that you know, we can talk about it now or if you we
- 3:13can talk about it later, allows us to basically put that gene
- 3:18back into the retina of those patients so that we can address
- 3:21the the root cause of the of the disease.
- 3:23Yeah, understood. And that plays into the name of
- 3:26the company, right, Because it's quite a large mutation and that
- 3:28sure what your technology enable enables you to deliver that.
- 3:33Yeah, Station, whereas classic technology and gene therapy
- 3:37might. Yeah.
- 3:39Exactly. So our technology, it's based on
- 3:41these reaction called protein splicing.
- 3:43That's what we were studying at Rockefeller and Biansten.
- 3:46There was a reaction that, you know, back in the day, they
- 3:48didn't really make it into the textbook biology books.
- 3:51So many people know about RNA splicing, but DNA protein
- 3:55splicing, it's a little bit more sort of obscure and basically a
- 3:59reaction that from 2 protein fragments we can generate the
- 4:03full length protein from those bragments.
- 4:07And this is catalyzed by some smaller sort of molecular
- 4:10machines that we that are called indanes, men are act as sort of
- 4:14molecular Velcro. So in starter patients, they
- 4:17have mutations in the Super large protein.
- 4:19So typical gene therapy approaches that are based on the
- 4:22use of viral vectors to deliver those genes into the retina of
- 4:26patients don't work because the gene is too large, so you cannot
- 4:29fit it into those vectors. And so our protein splicing
- 4:33approach allows it. We've basically split this 2
- 4:36into two fragments. So we can call the liver 2 viral
- 4:39vectors. And then inside the cell,
- 4:41through protein splicing, we can form the full length of protein
- 4:44notes. So yes, the name of the of the
- 4:46company, I think it's a good reflection of what we actually
- 4:49do. We do splice, yeah.
- 4:51Yeah, 100%. There's always a story
- 4:53somewhere. Yeah, yeah, yeah, yeah, yeah,
- 4:55yeah. Absolutely.
- 4:56Awesome. Cool.
- 4:57Where were you guys up with things currently when it comes
- 5:01to the the clinic? Walk us through that journey a
- 5:03little bit. So as you know this year, but
- 5:05we, we raised our Series B financing, we raised you $135
- 5:10million, which you know, allows us to basically take our program
- 5:15into, into the clinic. So we dosed our first patient
- 5:19earlier this year. As far as we know, it was the
- 5:21first time someone had put in things into, into human beings.
- 5:26So I think it was a huge, big step for us and, and hopefully
- 5:30for, for the field. And you know, the trial has been
- 5:32progressing very smoothly throughout this year.
- 5:35And we're gearing up to continue with the trial and, and, and you
- 5:39know, start to treat more patients so that we can in the
- 5:42coming years generate safety and efficacy data to to allow us to
- 5:46demonstrate that that this therapy works in humans and can
- 5:50address the, the unmet need in the patients.
- 5:53So if you don't mind, just walk us through the, yeah, the
- 5:55timelines you mentioned obviously, but dosing the first
- 5:59patient in your phase one trial and when are you expecting to
- 6:03know more about the outcome of of Phase 1?
- 6:06Is that next? Year or so we the plan is that
- 6:09we the financing of a serious Bay will reach a clinical proof
- 6:12of concept and we're doing well to to achieve that.
- 6:17Yeah, the airlines done. Yeah, sure.
- 6:20And that's, yeah, pretty key, right.
- 6:22You've got to be capital efficient to get to your next
- 6:25inflection point, get to the next day, which allows you to,
- 6:28yeah, raise more capital and to get key cycle.
- 6:31Yeah, exactly. Yeah, exactly.
- 6:32To get to the next. Stage, Yeah, no, it's exciting
- 6:34though, for sure. Cool.
- 6:36I mean, look, we touched on as well during our our prep for
- 6:39this that you know how key Barcelona has been for the
- 6:43company, obviously, yeah. In my hometown.
- 6:46Yeah, yeah, yeah. And, and Sylvie's as well,
- 6:49right. Yes.
- 6:50So yeah, it's definitely not a a basil or.
- 6:56Yeah, it's not Boston. Yeah.
- 6:58A Boston yeah exactly I was just thinking European but yeah it's
- 7:02it's definitely growing right there's has to be a lot of
- 7:05success stories coming out of it I know it's an obvious reason
- 7:09why you start the company sure right yourselves until this bus
- 7:13was there other factors involved in that and.
- 7:16Yeah. So I think, you know, both
- 7:18Barcelona and Catalonia and more generally are doing things well
- 7:21in India's base to try to, you know, grow this biotech sector
- 7:27as a, as a, as a, as a sector of the economy that the, you know,
- 7:31the region wants to strengthen. For me, as you said, they were
- 7:34obviously personal reasons of wanting to go back in a way it
- 7:37had received, you know, throughout my academic career,
- 7:41numerous fellowships from, from public funding from the
- 7:44government, etcetera. So to me, when we decided to
- 7:46start the company, there was also a little bit of a yeah,
- 7:50idea of being able to contribute or sort of give back to a sector
- 7:54that was starting at that point. I think Barshana and Catalonia
- 7:58are doing things well. It's a, it's a very good place
- 8:01for basic research. So there are great research
- 8:04institutions where research of excellence is being conducted.
- 8:08I think there's also very good hospitals for clinical research.
- 8:11I think sometimes, you know, we decided that in Spain, it's that
- 8:14you're the country where the most clinical trials are run in
- 8:17Europe. And Barcelona is a city within
- 8:19Spain where the most clinical trials are are run.
- 8:21So there's a lot of experience in running clinical trials.
- 8:25And then on the private end, I think there's a quite active VC
- 8:29community. So many of the VC funds in Spain
- 8:33or South of Europe are actually based in Barcelona.
- 8:36And culturally, I think there's this stereotype, which I think
- 8:39it's, it's, it's partially true as all stereotypes that Catalans
- 8:44have these strong spirit of entrepreneurship.
- 8:46So I think that gives also the, the spark for people to want to
- 8:50start new ventures. And I think that's creating a
- 8:52little bit of like broth from good things can happen.
- 8:57And I think that this the, the, the Bartian habits definitely
- 9:00growing and we are seeing more and more.
- 9:02Hopefully we'll continue to see more.
- 9:05We just need to complete the cycle, right?
- 9:06We're seeing good financings, we're seeing deals.
- 9:09We need to start more to see more and more good exits as
- 9:12well, as well as drugs getting into the market, right?
- 9:15That's the final goal for for all of our companies, yeah.
- 9:19I know you touched on it, but the the VCs in the area are
- 9:22quite focused on those early stage investments.
- 9:26Yeah. So many of them our strong
- 9:29company creation and early stage investments, but they also do
- 9:32obviously investments in late stage.
- 9:34And I think many of them. And the first one that started
- 9:37to do that was kind of one of the earliest funds was Issios
- 9:41Capital who did our seat financing.
- 9:44And they were from the early days very active in also
- 9:47investing outside their turf, right, See investing in other
- 9:51European and even American companies.
- 9:53And I think that was a necessary step to, to kind of showcase
- 9:58what's being done locally, which then enable, you know,
- 10:02international investors to eventually come back and invest
- 10:05also in personal companies. I, I don't like to put ourselves
- 10:08as an example of anything, but it did happen in our case right
- 10:12where we our Series A was called by ECS, but UCB Ventures and we
- 10:17were able to bring many other investors including Gilden and
- 10:21Novartis went to fund, but also NEAUS investor that had not
- 10:25previously invested in in Spain. Yeah, no, for sure.
- 10:28I think he did a podcast with Geordie right around assignment
- 10:31to CSPS. Yeah, yeah, that one is in in in
- 10:35Spanish. So I don't.
- 10:36Yeah, how how I think, Yeah. And the Spanish, that's three
- 10:39sub at idols. Yeah, I'd have to get an AI
- 10:41translator or not for myself, but no, Yeah, How, how did you
- 10:46find raising the series being this market?
- 10:48Because earlier in the year, things were not down in the
- 10:51doldrums, but they were difficult, right.
- 10:52I think we've seen a little bit more positive traction in the
- 10:55past few months. But yeah, when was?
- 10:58It that you announced a series. Based some, so we announced in
- 11:00the summer, in the early summer and it obviously, yeah, it it
- 11:06was tough to raise it because obviously you're announcing the
- 11:08summer that means you've been working on it for a while.
- 11:11Yeah, actually some of we start we knew it would it was a tough
- 11:16time to be fundraising. So we actually started very
- 11:19early on to start having those informal contacts with investors
- 11:23even before you are formally fundraising.
- 11:25I think we had one of those initial meetings with actually
- 11:28our who eventually led the round EQT.
- 11:31We had one of those first meetings at Jeffrey's 2023.
- 11:39It was in a coffee shop here next to the Waldorf and we met
- 11:43with Daniela, who totally became because of the lead from the EQT
- 11:46side at that time. Just if you know, getting to
- 11:50know each other, but that just then you that relationship grows
- 11:54and the process moves forward and then eventually they, they
- 11:57become the the lead investors. But yes, it's, I think it's
- 12:01still taking longer than obviously in the 2021 time
- 12:05frame, but I think it is getting a little better and we're seeing
- 12:08some more financings and that helps, you know, everyone to
- 12:14become sort of more active. Yeah, you know.
- 12:16For sure. I think you hit the nail on the
- 12:18head. It's time, all right?
- 12:20And yes, yes, yeah, CEOs, most of the zeros in our network are
- 12:24always fundraising. That might just not be
- 12:27officially fundraising. They might not be in the process
- 12:29of actively raising but those conversation.
- 12:33Need to happen and obviously, and I think I've mentioned this
- 12:37in in other interviews or party has the importance of trust and
- 12:41it's not that secret sauce, but you know trust takes time for
- 12:45Shan and and they need to see you also executing and they
- 12:49typically they'll never have enough with the data package
- 12:53that you presented right. There's always something else.
- 12:56So you did and that's the same with public financings, right?
- 12:58You need to have like something else coming up that they can
- 13:02that can become the catalyst for, for the financing.
- 13:05Yeah. And in our case, it was the Ind
- 13:08filing. Yeah, for sure.
- 13:09How have you found the whole yeah process of, of, of
- 13:14fundraising, but also being ACEO from a scientific background?
- 13:19Yeah. I mean, it's, yeah, it's a
- 13:21different world, right from the from the research side of
- 13:24things. So yeah, how's that
- 13:26transitioning? For you now I've been it for a
- 13:28while. So I I managed to partially
- 13:32forget or, or or put on my back the the the in imposter syndrome
- 13:37is that I had in the early days and sometimes it's still like
- 13:41creeps out saying I think. I think as a scientist, you are
- 13:45trained to constantly having to learn new things.
- 13:48And that's definitely that I can relate as a, as a CEO as well,
- 13:51right? At least of a small growing
- 13:53company, you're constantly faced with new challenges that you've
- 13:55never faced before. So you really need to have the
- 13:58ability to, to learn and, and be willing to, to learn and be
- 14:02willing to admit that you don't know about that stuff and you
- 14:05need people that will help you learn about it.
- 14:07So I think that's something that I can relate with the two paths,
- 14:12maybe something that it's challenging as a as a CEO and
- 14:15maybe as a scientist or this. I was trained within my project
- 14:19to basically take control of everything, be the master of
- 14:23everything as ACEO. That's not possible.
- 14:27But at the beginning there is the tendency to do that.
- 14:30So there's the risk of micromanaging the team.
- 14:33So that's something that you need to learn how to delegate
- 14:35effectively in a way that things can happen.
- 14:38And then you also feel comfortable with that
- 14:40delegation. So that I think now I mean a
- 14:43good place, but that's been a learning curve.
- 14:45Yeah, is that the advice you pass on to someone in a similar
- 14:49position or looking? Yeah, yeah, yeah, definitely
- 14:53find a way that you can delegate efficiently and then obviously
- 14:57try to surround yourself with the best people that you can,
- 15:01right. There's this half reason that I
- 15:03don't know, we said it about, you know, if you are the
- 15:06smartest person in the room, you've brought, particularly as
- 15:09a CEOI think you've done something wrong.
- 15:12I think you should have strive to have people way smarter than
- 15:15yourself helping the company move forward because I mean,
- 15:19we're definitely just yeah, yeah, there's enough in our
- 15:21shoulders. So you really need to have a
- 15:23good team. And I think we've been very
- 15:25fortunate. No, definitely I supplies to.
- 15:27Be able to build. That thing, yeah, delegating
- 15:31absolutely key, But you can't delegate to a poor team.
- 15:34You have to have excellent people, yes, around you, right
- 15:37as the adage of of hiring smarter PDF than yourself is.
- 15:41Yeah, definitely. Yeah, definitely true.
- 15:44And sometimes it's not intuitive, right?
- 15:46Because I think particularly when you're technical experts,
- 15:48you're used to it's comfortable, comfortable to lead from the
- 15:53excellence that you have in a specific field.
- 15:56Yeah, but that's not the only way to lead, right?
- 15:59And you need to be comfortable leading people who as who said,
- 16:02right, They are smarter and they know way more than you about
- 16:05certain things. But otherwise it's impossible.
- 16:07Yeah, no, for sure. I mean just looking super long
- 16:10term and and not all in you to anything.
- 16:12And you know, feel free to say what you you can get and will.
- 16:16But like long term, what's the what's the vision for where,
- 16:20where you're taking things like roughly in the space?
- 16:24Are you looking at just the Sjogren's?
- 16:26Are you looking at other indications?
- 16:28What's? What's the thinking?
- 16:29No. So I think obviously our central
- 16:32focus, it's developing the the treatment for Stargart.
- 16:35There's a clear unmet need. There's patients that are sort
- 16:38of waiting for, for these therapies.
- 16:41We as a company, we have this technology that really allows us
- 16:44to develop treatments for many diseases.
- 16:48There's many diseases that have this limitation that you cannot
- 16:51put them in a small viral vector.
- 16:53So there's this vision of, you know, really through our
- 16:56technology unlocking the potential of gene therapy in all
- 16:59those diseases. We internally we're focusing on
- 17:03tissues where we can do localized administration and
- 17:06that's definitely the eye and it makes a lot of sense for the
- 17:09technology. And I think we see also it makes
- 17:11a lot of sense from the sector perspective.
- 17:13We see a lot of fundraisings in that space.
- 17:16We see a positions. So I think the field it's honing
- 17:20on that for for a reason and for geotherapy and for us.
- 17:25So that's an area of interest beyond ophthalmology.
- 17:28I think the CNS or the inner ear, other organs were
- 17:33obviously, you know gene therapy.
- 17:35I think it's a, it's a modality that makes sense and where our
- 17:39technology can again, as we said, unlock many targets that
- 17:43are not possible, we ought our and approaches.
- 17:46Why do you think? I mean, I guess there's a few
- 17:48different reasons behind why ophthalmology has been a little
- 17:51bit of a safe haven for GFA because yeah, on a wider basis
- 17:56like that there's even first half scientists in Chesters this
- 17:59year, right. But ophthalmology focused
- 18:01companies seem to be pulling through.
- 18:03Yeah. Do you think that's purely
- 18:04because it's, Yeah, localized oil and safety issues, less, not
- 18:09non existent, but yeah, less, yeah, that's pressing or?
- 18:12Yes. So I think there's a couple of
- 18:14the key challenges EG therapy are the safety aspects and the
- 18:19manufacturing. I think in ophthalmology you
- 18:25basically just by going in that organ we take care of those two
- 18:28problems off the bat, right. So obviously doses are within
- 18:32thousands of times lower than what you need for systemic.
- 18:35So in terms of the manufacturing, it makes the
- 18:38whole thing much easier. It also takes care of the
- 18:41economics of cost of goods etcetera.
- 18:43So which it's an issue for other for systemic gene therapies in
- 18:47ophthalmology it's a non issue. And then as as you mentioned
- 18:51raised there's the also these immune privileged or partially
- 18:55immune privileged nature of the eye, which also makes the the
- 18:59safety profile much more. Yeah, there is benefit might get
- 19:03a favorite. Yeah, got you.
- 19:05So, and I think, you know, we've seen in the, in the past years,
- 19:11maybe in the 2020-2021, there was this huge expectation that
- 19:15gene therapy and maybe gene editing would be the cure for
- 19:18all diseases. And that's something that then
- 19:22maybe it was faced with a reality and there was like a
- 19:24really negative reaction to, to the space, which was probably
- 19:28maybe also an overreaction. And I think now we're getting
- 19:32two more mature, stable positions where gene therapy
- 19:36will be suitable for some therapeutic areas or for some
- 19:39indications. The fact that it doesn't need to
- 19:43work for everything doesn't mean there's no value.
- 19:45Yeah, yeah. In the.
- 19:46Modality, that's fine. I think as humans we have a
- 19:49tendency to overreactive things. And yeah, like some bees,
- 19:53amazing one minute and awful the next.
- 19:56Like modern society particularly has that problem.
- 19:59I mean moving forward for gene therapy.
- 20:03Generally upside of the Where do you see the field going in the
- 20:07next few years? Yeah.
- 20:08So I think the key it's that no, I think it will find after this
- 20:13happens at some downs, I think it will find its place amongst
- 20:17the arsenal of modalities that we have to treat diseases.
- 20:21And I think we see a more focused approach to gene therapy
- 20:25where it will not be this huge promise of it working for
- 20:28everything. It will be a more focused effort
- 20:33in the areas where gene therapy makes the most sense at the
- 20:36current time, right, Because there might be developments in
- 20:38the future where then it can be expanded in other areas.
- 20:43And, and I think one example, again, it's our technology,
- 20:45right? For a few years ago, you could
- 20:48not even consider to go after these larger genes because there
- 20:51was no way to deliver them. And I think our technology now
- 20:54opens the door to go after those genes.
- 20:56But I think overall there will be room for gene therapy again
- 21:00for, you know, not again, but in the sense that, you know, again,
- 21:03as I was saying, yeah, there will be room for, for, for gene
- 21:06therapy as a modality. But we will need to really focus
- 21:10on where it makes sense, not just purely because it's a cool
- 21:13technology. We need to use it for for
- 21:15everything. Yeah, I get you and I like that
- 21:18it has to fit into the arsenal of what else is out there,
- 21:21right. It's not a one stop cures all.
- 21:24Yeah, exactly, exactly. And and we see that, right.
- 21:28Obviously when you talk at the end of the day, the patients
- 21:31don't care about which technology you're using to treat
- 21:35the disease. And but biotech and particularly
- 21:39I think gene therapy companies because they are so tech heavy
- 21:42and sometimes there's been this trend to see everything through
- 21:46the lens of the technology. Yeah.
- 21:47And at the end of the day, what matters is you know, which
- 21:50disease you're trying to cure. Is your approach the suitable
- 21:53for that disease? Yeah, not if it's gene therapy,
- 21:56gene editing, a small molecule or an antibody.
- 21:59Yeah, I think that's an important thing for everyone to
- 22:01remember about. Yes.
- 22:02And it really doesn't get all they just want to be fixed as
- 22:06entries survey. Yeah, because notes finish on
- 22:09for sure. No.
- 22:10Thanks for coming on, Michael. Really appreciate it.
- 22:12We'll finish with the standard biohop question, which we
- 22:15thought it's about beforehand. The.
- 22:17I think I may know the answer, but yeah, that's if he could
- 22:20have dinner with one scientist, past or present, or an
- 22:23entrepreneur if you want. Who would it be and why?
- 22:28OK, Yeah, maybe. Yeah.
- 22:31I mean, the scientists have come to mind.
- 22:32I mean, he's not maybe not very well known to the general
- 22:36audience, but there was a Nobel laureate called Bruce
- 22:38Merrifield. He was the inventor of peptide
- 22:41synthesis. So that's my background.
- 22:43My background comes on how to synthesize peptides.
- 22:46He developed a method that really, it's then the method
- 22:49that has been used, also adapted to synthesize oligonucleotides
- 22:52that it has really made possible, the genomics and
- 22:55proteomics revolution. And yeah, it'd be cool to talk
- 22:59to him to see if, you know, he envisioned the impact he would
- 23:02have. Yeah.
- 23:04Based on that. So I think, yeah, I think it
- 23:06would be good to have so dinner with him.
- 23:08He's my sort of academic grandfather, so there's that
- 23:12relation, but I never had the chance to to have the dinner
- 23:15with him. It's.
- 23:16Nice, nice, good answer. Sure.
- 23:18Cool, Awesome. Well, great.
- 23:19Great to see you. Likewise, thank you for having
- 23:22me.