Latest / The BioHub - by Avetix / Josh Mandel-Brehm - CEO CAMP4 Therapeutics at BIO Boston 2025

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Josh Mandel-Brehm - CEO CAMP4 Therapeutics at BIO Boston 2025 · The BioHub - by Avetix

• 0:04
  So here we are at Bio in Boston. I'm with Josh Mandelbraham.
• 0:08
  He's the CEO of Camp 4 Therapeutics.
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  Just give us a bit of an introduction to to who you are,
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  Josh. Yeah.
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  Thanks for having me, Max. You nailed the name, so well
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  done. As Max mentioned, I'm the CEO of
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  Camp 4 Therapeutics. At Camp Four, we use antisense
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  algonucleotides to increase the expression of genes.
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  So I like to tell people think about us as the inverse of Ionis
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  and on Iowa in the sense that we can make things go up instead of
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  making things go down. OK.
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  And why that's important is it turns out that there's hundreds
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  of diseases where you're missing healthy protein.
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  And so if you can find a way to put healthy protein back in the
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  body or teach your your body how to do more of what it already
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  knows how to do, that is make more protein, that could be a
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  hugely valuable new form of medicine for a lot of different
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  patients. Yeah, that's amazing.
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  And we were just talking about how you guys are so different to
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  what what else is out there. Just just walk us through that.
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  Yeah. You know, one of the things
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  about Camp 4 is we've we've always done things differently,
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  not for the sake of it, but because we've had to.
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  So, you know, many times in in company creation, you see
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  there's Nobel Prize winning science or there's a big step
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  forward in innovation, CRISPR as an example.
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  You see many companies come up, which makes a lot of sense.
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  Camp 4 is a little bit different in the sense that we had a
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  technology that allowed us to essentially map cells, that is
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  go in and look at what's happening in the nucleus of
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  cells, how the genome is making decisions to control its genes.
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  But the question for us was really how do you use that
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  information to make drugs? And by working very closely with
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  our founders, Rick Young and Lens on what we discovered at
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  Camp 4 is that there's a new category of RNA is called
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  regulatory RNA. So RNA is that come out of
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  enhancer and promoters the non coding genome that every gene
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  uses to control its expression. So essentially, as Rick would
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  say, Doctor Rick Young, you have to rethink the entire rules of
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  gene regulation. And so that's really exciting
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  for us. But the point is we're
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  pioneering this new area of upregulation using ASOS at the
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  same time that Rick in the field are pioneering gene expression.
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  And so that's a little unusual. Typically companies come after
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  that happens, but we've been doing it in parallel.
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  And I think that's pretty exciting because as a science
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  advances, we don't have to wait to make new medicines.
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  By using anesthesoligos, which are a great type of chemistry
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  that have been around for many decades, we can actually make
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  hay and start making drugs while still understanding the science
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  as we go forward. When you are that novel as well,
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  what's it like talking to investors and how do investors
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  react to that? Yeah, so there's definitely an
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  element. There's two things to think
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  about. 1 is that it's important to really simplify the story.
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  Not simplify to miss the details, but simplify to help
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  people really understand what's most important and then you can
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  get into the details later. So, you know, as this is a newer
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  field that has been, it's taking time to educate people on that.
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  Some of the terms we use are not textbook terms.
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  So we've had to build that over time.
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  The other thing is as you evolve as a company, you know, we, I
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  started a camp for with five employees, you know, now we're a
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  publicly traded company and so the types of investors we talked
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  to evolve over time too and how they think about the company and
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  what's important to them. So it the company matures, how
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  we talk about our science changes depending on who we're
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  talking to. And we're certainly in a world
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  now where what we really care about is we think we can make
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  medicines for diseases that where there's no treatments and
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  there's big opportunities. And I think that's really the
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  punchline. What a.
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  Journey as well, five people to what you are now.
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  It's just walk us through what that's been like.
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  Yeah. Look, we, we've, we've had to
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  make, you know, I think all great companies in and outside
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  of biotech have sort of life saving moments, if you will, and
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  have to make really hard decisions and those same hard
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  decisions become opportunities that help you become an even
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  better company. And, and Camp 4 is no different.
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  So our first iteration of the company when it was founded in
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  2016, Seventeen was using that mapping technology, but then
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  also using small molecules. And we raised money around that.
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  We did some partnerships, but we found that the combination of
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  our technology with small molecules wasn't specific enough
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  for the types of diseases we wanted to go after.
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  Yeah, it was being rare genetic diseases.
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  And so we shifted based on some of the biology coming out of
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  Rick Young's lab. And it was kind of a daring
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  moment for us. We did that in 2020 and we
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  started using oligos to increase genes and we haven't looked back
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  since then. Since then, we've been able to
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  really move from testing in a in in vitro Petri dish setting in
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  2021 to now wrapping up our first clinical study or close to
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  wrapping it up in humans using new technology.
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  I think that's pretty exciting and it shows how quickly we can
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  move with this type of biology. So how often do you actually sit
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  back and go like we, I know there's a long way to go, but
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  look how far we've come and be super grateful for that.
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  In this in this business, I have found there's more lows and
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  highs, but you've got to turn them into opportunities and we
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  try and really savor the high moments.
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  I started my career working at bigger companies, Genzyme,
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  Sanofi, Biogen. And if you had told me that
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  later in my career I'd be really excited about some exciting
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  mouse data or primate data, I would have laughed.
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  But, you know, those are great moments for us when we see our
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  technology can do something unexpected in a monkey, which
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  should translate to humans. When we got our first drug
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  approved to go into the clinic, when we put our drug into a
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  human being and show that it was safe.
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  I mean, those are incredible moments that we've worked hard
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  to get to and we certainly try and relish them.
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  I I like to say we take a day to celebrate and then we get back
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  to work. Yeah.
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  Nice. And then?
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  We're like. The culture piece, right?
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  You've gone from 5 people, you've grown, you've become a
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  public company. Company obviously evolves in
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  some way. Like how do you really map
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  culture, define culture across the the company?
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  Well, I, it's a really important question.
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  I think every CEO thinks a lot about it for their companies.
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  My, my view is culture is a set of actions and virtues.
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  It's not values. I think people sometimes confuse
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  that. And what I mean is values are
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  things you write on the wall. Culture is a set of actions and
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  how people operate day-to-day. And so we think very long and
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  hard about, OK, what are the things that we do that make us
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  us? Yeah, that's part of our culture
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  and one of the things that we aspire to do.
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  So as an example, we do a survey every year where we ask the same
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  question, which is, do we have a culture?
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  And how would you describe our culture in one sentence?
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  And the Arrow Bar should be pretty tight around that.
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  We do that every year. And so if you were to do that
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  today, what you'd hear across the company is it's, it's high
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  science, it's fast-paced, it's an extremely transparent
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  culture. Despite the fact that we're
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  public, there's a lot of collaboration.
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  And those are things that one, we, we operate that way and we
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  try and build around. So one of the things we do, we,
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  we have lunch for everybody on Tuesdays and Thursdays, not just
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  that for the sake of having lunch, but because that brings
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  people together, right? Because that's part of our
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  culture. We operate in certain ways
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  where, you know, I'm just finishing up where for the next
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  few weeks I'm having coffee chats with the entire company,
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  but in small subsets, 5 to 6 people.
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  Because when you're in a big setting, people don't
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  necessarily want to talk, right? You got to get people talking.
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  That's an element of our culture.
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  And by the way, cultures can evolve.
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  So as we, you asked the question, we've gone to five now
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  to 50. What's changed?
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  Well, there's some elements that haven't changed.
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  High science, fast-paced, collaborative, our transparency,
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  how we communicate, how we develop people.
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  That's shifted in terms of our culture, but we're very
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  deliberate about it. And I think as I've told my
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  employees, which is culture is not me telling everybody how we
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  have to be, but it's also talking with them about how they
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  want to be. And if you want to operate a
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  different way, then you actually have to change your behavior.
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  We can do that. But where culture really
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  matters, of course, is when times are tough, when the tide
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  goes out, you know who's skinny dipping?
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  And so for us, it's like that's when culture really shows up and
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  that's where the opportunities come.
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  And I think this company has lived through some really hard
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  moments and not only live to fight another day, but come out
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  really stronger on the other side.
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  So I think it's a very resilient culture and one where the people
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  are at this company because as we talked about in the
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  beginning, what we do is different.
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  Yeah, we feel a bit like we're pioneers in that sense.
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  No, I love that and will completely change the topic now
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  just because it is a big topic in the industry.
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  Pub being public versus being private.
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  Yeah, especially in the current market.
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  Yeah, I'll leave that question. Broad look, so I'm fortunate
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  enough that I've gotten to live on both sides of this now.
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  So we're private I did 4 private fundraisers.
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  What I can tell you is they were never easy not not because you
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  know people didn't believe in what we're doing or what not but
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  we had to prove to them what we were doing mattered and each
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  step that took time and we got the investors we deserved, which
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  were great investors and they really are partners to us and
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  helped us build this company. What I can tell you about the
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  benefits of being private are you get more flexibility, so
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  your cap table or your ownership is concentrated usually to major
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  investors. They're on your board, you're
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  having discussions with them on your board, but you're making
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  those decisions together and you could change them relatively
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  quickly, but that's who you're answering to, That's who you're
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  having a dialogue with. Now, when you're public, you
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  have a lot broader set of shareholders.
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  You're public, You have to earn it every day.
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  Yeah, every single day you have to earn it.
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  And you have a scorecard every day, which is your share price.
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  When you're private, you only get your scorecard where you
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  raise money. So there's a psychological
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  aspect to it. The one thing I'll say about
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  being public, which I think is a massive benefit, and I say this
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  having lived on both sides now, is being public offers you
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  access to a broader set of shareholders and investors and
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  also different vehicles to raise capital and a more efficient way
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  to raise capital. Right.
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  So fundraising becomes a bit more process oriented because
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  public investors are looking at hundreds of things.
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  Yeah. And they're not taking, they're
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  not doing 3 to 4 investments a year.
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  Private investors are, they're, they're more concentrated, they
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  take a lot more time. So a private fundraise can take
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  anywhere from 9 to 12 months. That is a lot of work for you
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  and your team. It can be a big distraction, but
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  it's what you need to do. It's just the way the world
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  works right now. Public investments don't work
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  like that typically. It's a lot more efficient.
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  So each of them has trade-offs. And I think we're as a company
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  in the definite right place to be public.
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  I think it's the right thing to us.
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  And I think we just have to earn it every day.
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  And I think that actually it's a benefit being public, and we
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  understand the responsibilities that come with that.
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  But we were private for seven years, so it was time for us to
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  take that next step. Yeah.
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  Yeah, definitely. And what, what about what's
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  next? What's exciting?
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  Yeah. So look, we, as I mentioned to
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  you, I think what the most I, I look, I have a lot of
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  aspirational people and companies we look at.
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  But if I just take one, we like online them as one because we
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  think what they did was really amazing.
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  But more so they showed that their platform can make more
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  than one product and that's what we're aspiring to do.
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  So we have our first program in the clinic that's reading out,
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  but we have our next program for a really exciting and the first
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  one's for urea cycle disorder. Our next program is for a rare
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  disease called syngap, which is a devastating disease with no
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  approved disease modifying treatments.
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  It's ACNS disorder. Why I'm really excited about
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  that one is if if you just look at the data that we have and you
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  look at these families, it's we're giving them hope.
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  And so we badly want to get there.
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  But then a lot of our technology behind that is going to be in
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  the CNS. And so I think there's a lot of
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  really great opportunities in front of us.
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  I think in the next two years, we're going to have readouts
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  coming and there's going to be a lot of exciting data cards
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  flipping over. And my view is that's when we're
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  going to show the world that this technology works.
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  And then I think things are going to really start shifting.
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  That's when you start taking the next step as a company where
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  when you show something from a political perspective, you're
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  now getting in rarefied air and you're starting to actually get
• 11:18
  more credit for what you're doing and you can start to do
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  other things as well. So that's what I'm really
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  looking forward to. I think that's what we've been
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  working for the past seven or eight years.
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  And, you know, I think we're all excited to get there.
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  We have more work to do. It'll never be fully linear, but
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  I think these are the types of diseases where there's really
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  nothing there. We can make a huge impact with
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  our technology. It's uniquely suited to do that.
• 11:37
  Yeah, I think that's a great place to leave it.
• 11:39
  Thank you so much for coming on. Great.
• 11:41
  Terrific.