Gene Editing With a Personal Mission

Puzzle pieces factor in today's episode, both about DNA, Duchenne, and perhaps we figured out the magic behind cool companies. Listen now. Welcome to The Connect Current, the flow of innovation in San Diego. We bring you the founders, investors, and leaders building the future across life science, tech, climate, defense, AI, and more. This is where San Diego's next chapter takes shape. Welcome to The Connect Current, the flow of innovation in San Diego. I'm your host, James Zanewicz, and this episode is being recorded at Kiln in Rancho Bernardo. We want to thank Kiln for their support and let everyone know that this is Connect's new second home. So please visit us at Kiln. Today I'm joined by Courtney Young, PhD, co-founder and CEO of Myogene Bio, and one of Connect's 2026 Cool Companies. Myogene Bio is developing cutting-edge genetic therapies for muscle diseases, with its lead program being a gene editing therapy for Duchenne muscular dystrophy, a devastating rare muscle wasting disease with no cure. Courtney's path into this work began in high school when her cousin was diagnosed with Duchenne. That diagnosis led her to muscle research as an undergraduate at Johns Hopkins, master's at UCL, and then a PhD at UCLA, where she developed the gene editing approach behind MyoDye-4555 alongside her advisors, Dr. Melissa Spencer, and Dr. April Pyle. The 3 of them went on to co-found Myogene Bio in 2018, spinning the company out into UCLA's Magnify Incubator before relocating to San Diego in 2022. Myogene Bio is also notable for being founded and led by 3 women scientists, something Courtney has spoken about as still uncommon in biotech. And the company has drawn support from organizations including Cure Duchenne Ventures, How Women Invest, and Parent Project Muscular Dystrophy. This is a great Connect Current conversation because because it brings together gene editing science, a deeply personal founder story, and the real work of building a life science company. It's also a great example of why we're excited to recognize Myogene Bio as part of Connect's 2026 Cool Companies. Courtney, welcome to the show.

Thank you so much for having me.

We're excited to have you here, but we may have some listeners who aren't familiar with you or Myogene Bio yet. So can you tell us briefly, what are you building? What problem are you solving?

And what makes your AAV-delivered CRISPR approach to Duchenne Myogene is a biotech startup that's really interested in developing novel approaches that aim to save the lives of patients with muscle diseases. Our lead program is a gene editing therapy for Duchenne muscular dystrophy, which is a genetic disease caused by mutations in the dystrophin gene. And these mutations basically cause the puzzle pieces of DNA to no longer fit together. That makes the gene nonfunctional, and so no dystrophin protein is able to be produced, which would normally be protecting muscle cells. And so in Duchenne, patients succumb to progressive muscle degeneration and ultimately death. Our approach uses CRISPR gene editing, where we use molecular scissors to go in and cut out a region of the DNA. So this will put the puzzle pieces back together again so that the dystrophin protein can be restored. Fixing the underlying cause of disease in Duchenne. We specifically have designed our approach to target a hotspot of half of all patient mutations. So with one approach, we can target a large proportion of patient mutations and mimic a very mild disease course observed in humans, aiming to make a functional dystrophin protein. And so the benefits of a gene editing approach compared to other approaches in Duchenne are that we're permanently editing the patient's own DNA, in contrast to approaches that are transient or aren't permanent. We're keeping the natural So the protein is expressed how it normally should be. We're targeting this hotspot, so half of all patient mutations, in contrast to approaches that just target one exon, which would be at most applicable to 13% of patients. And then because we're using AAV to deliver CRISPR and not the gene itself, which is very large, we're able to restore a dystrophin protein that's almost 90% the size of normal, in contrast to some of the gene replacement therapies that have to deliver the gene, which doesn't fit, and so they make a miniature version of it. version, it's about a third the size of normal. So we're able to make a 3 times larger protein aiming to be more functional in Duchenne.

So to personalize this a little bit, how would you describe what Duchenne does to a family for someone who's never heard of it before?

In about a third of the cases, Duchenne is a spontaneous mutation in that child. So there's no family history a third of the time. And that means in cases like my family, we had never heard of Duchenne before. It's still a rare disease, affects about 1 in 5,000 newborn boys and You know, it's a very progressive disease. So what happens with patients is that they lose muscle over time. Eventually, patients lose the ability to walk. They end up in a wheelchair, unable to lift their arms very high, and then succumb to death due to cardiac or respiratory failure, typically in the 20s or 30s. But there's been some advances in helping to prolong life now. When my family member was diagnosed, there were no approved therapies for Duchenne, and basically there was nothing you could do. And so the field has now come a long way where we're beginning to have a lot more research and interest, and now some approved therapies. Yeah.

And you shared how this has all started for you when your cousin Christopher was diagnosed in high school. How did that moment carry you from studying muscle biology into eventually deciding to found a company that could improve lives for people like him?

Yeah, when I was in high school and my cousin was diagnosed with Duchenne, I had always been interested in science and math and engineering, but I didn't really know what I wanted to do. So I, when I went into undergrad, I began working in a muscle biology lab doing research on muscle and diseases like Duchenne, and I became really interested in it. Muscle is a very important tissue. There's a lot going on, and I spent some time working on various therapeutic aspects for Duchenne, ranging from small molecules, exon skipping, stem cells, things like that, and realized that to have a truly meaningful impact on the disease, I wanted something that was going to target the cause of the disease.

Mm-hmm.

And so going in to kind of fix those genetic mutations would be really impactful in a disease like Duchenne where there's a known mutation, there's a known gene. And that's what got me interested in the idea of gene therapies. And I went to UCLA for my PhD specifically because there's a whole group of researchers working on Duchenne there. And I joined the lab of Dr. Melissa Spencer with a co-advisor, Dr. April Pyle. And this was right when CRISPR had been published for use in human cells. Mm-hmm. And we said, you know, everyone's talking about this new thing called CRISPR, maybe we should try it for Duchenne. We have no idea if it'll work, but I can spend a little bit of time and see if there's any potential. And it began to look so promising, it turned into my thesis project to develop a CRISPR therapy for Duchenne. And when we patented it and the data was looking really good, we said, you know, to really get this to patients, we think we should spin it out into a company. That's how you can develop a drug. I'd always been interested in entrepreneurship. My parents had done some entrepreneurship work.

Oh.

Not in biotech, but still, I had seen how they had been able to kind of build and direct a company. And we said, that'd be really cool to be able to take this pet project And develop it how we think it should be developed to bring it to patients. And that's what started the idea.

Yeah, I remember the CRISPR rage. That was back when I was at HHMI, and like, it was everything. It was the HHMI, the Allen, a whole bunch of groups came together for that. And so did a lot of collaborative phone calls and things like that. So I love to see the companies that have come out of that, because at that point it was just a lot of, not to say just talk, but a lot of talk about the science and seeing the science now, just what, a little over 10 years later, maybe 15, really coming into its own. But it's not just research. What Myogene Bio is doing is moving a gene editing therapy towards the clinic. And that comes with all the technical, regulatory, and manufacturing considerations, which make the science, you know, super challenging sometimes, but also exciting when you overcome that. And you're building that company alongside 2 fellow women scientists, which is still rare in biotech. What has been the hardest about turning this from a PhD product into a scalable company for you?

In the beginning, transitioning from academia into a startup, it's a whole different language.

Mm-hmm.

That you have to talk about, and you talk about business, and you talk about science in a very different way. So I was used to giving scientific presentations where you go deep into the science and the data. In the beginning, I think the biggest learning curve was just how to pitch your story to investors and tell it as a business story rather than as a science story. And so that definitely took some practice and a lot of advice from people, a lot of support from people who had done it before and were kindly to give a lot of feedback on the sort of story that we could tell to make it more accessible to the layoff audience and also to investors and talk about kind of how it could turn into a feasible business and not just a science project.

Hmm.

I think the goals in the company, of course, are to develop a therapy to bring it to patients, which is different than in academia. So kind of how we think about the experiments we do and how we would direct our time has shifted a bit towards developing drugs rather than just understanding biology. The underlying work, I think, has still been very similar. Directing a lab has been very similar, but I think that a lot of the initial lingo and learning how to pitch was the initial learning curve we had to get over.

Yeah. And gene therapies do face that long road from promising science to something that patients and families can access. Where are you seeing companies like MyogeneBio most often get stuck on that path, and how are you thinking ahead about that so that you can overcome those hurdles yourself, or how did you overcome them already if you feel like it's something you've already done?

Yeah, there's a lot of hurdles in gene therapy. I think—

It's hot again, at least, because for a moment it was hot, then it wasn't, and now it's hot again. So that's good, at least.

Exactly, exactly. We're at least beginning to get back into an uptick. But I think there's a lot of challenges. One of the quintessential ones is this valley of death between your initial demonstration of proof of concept to getting it into the clinic. So getting funding to pay for kind of the expensive IND-enabling and scaled-up manufacturing work, that can be a challenge. We've learned a lot from gene therapy clinical trials now about how to develop something with hopefully some better safety profiles while still getting efficacy. So I think having a well-designed clinical trial, and then of course getting reimbursement for the therapy, because gene therapies are different. They're typically just given once, and so they have a very high price tag for a single injection. Rather than, you know, a typical drug where you'd be having reimbursement over multiple years of dosing a patient. And so there's definitely been examples of therapies getting reimbursed, gene therapies getting reimbursed, but I do think as more and more therapies get approved, there'll need to be some innovation there. And then also hopefully innovation on the manufacturing side to drive costs down.

Mm-hmm.

So definitely there's a lot of challenges, but I think getting into the clinic, having a well-designed clinical trial, and then getting reimbursement and approval are all the kind of key ones.

Yeah. And once you get past those, you have to get that exposure and kind of that recognition for the company, for what you're building, which is why we're really excited that MyogeneBio is part of our Connect 2026 Cool Companies. But as you build this next chapter and have kind of your year of being a cool company halfway over, you have the 6 months left. What does being connected to the San Diego life science ecosystem mean for the company? And then as a second part to that, what kinds of partners, investors, or collaborators would be most helpful for you right now getting this into the hands of patients?

Yeah, well, first of all, thank you very much for having us as a Connect Cool Company this year. I think the San Diego ecosystem is one of the most friendly and helpful ecosystems I've been in. And so we've been really excited to be here in San Diego and just have everyone who offers to help or give you advice, but ask for anything in return, has been really nice. Right now we're working on getting through our preclinical development and planning our clinical trial. So in terms of potential partners or service providers, we're looking for groups who have experience with running tox studies in gene therapy and clinical trials in gene therapy. We have a planned Series A raise, which we're hoping to supplement with some grants to get through to clinical proof of concept, which at which point we'd be interested in potential pharma partnerships for kind of the later stage development and commercialization. Planning for both of those things and having networking conversations early is always great. But I think kind of just having an ecosystem of other company builders, people who have done it before, and support around getting a drug into the clinic is the biggest help right now.

And then very open to boatloads of cash and strategic partners.

Always.

I love that. Well, we always like to end on our very Connect Current note here, and that is our classic question of what drives your innovative flow?

Yeah, I think for me, it's always been an interest in taking a new technology and applying it in a way that would make a meaningful change for patients. There's been a lot of work in the field where approaches have been developed that maybe would just have some small effect, or they target a downstream pathway, and it's not going to be super transformative for the disease.

Mm-hmm.

And in a disease like Duchenne, which is so severe and progressive, It's really been my passion to have something that's really going to make a meaningful difference and really kind of change the trajectory of the disease, not just have slight improvements. And I think how you can apply new technology in those ways, especially ways that you can target the underlying cause of disease, I think will be really powerful.

Well, it's interesting because as we've had this conversation today, I was sitting here thinking in my head that I've been in many ecosystems, and you always find some stories with entrepreneurs that are driven by the deeply personal. But I feel like we have a disproportionate number And almost every single one of our cool companies is driven by that. Like, we've had many on this podcast, and I'm struggling to think of one that wasn't deeply personal. And I wonder if that's what makes you the cool companies that the investors that we bring in to look at you and kind of evaluate you for Connect and say, yes, these are the companies that are the top of the class. I think it's that personal spark, and it's something that kind of clicked in me today as we were chatting. And I just wanted to, to point that out, that I do think that is what makes you guys so special.

Yeah, I mean, I think you need a drive to be able to survive in a field like this. So if you have a personal connection to something, I think it definitely can make you be driven and successful and passionate about what you do.

Well, I want to thank you for having this conversation. I mean, it was really powerful about science. It was also powerful in being personal. I think it's a great reminder that the best science does come from personal experience and that Myogene Bio's work shows what can happen when that personal mission, rigorous science, and quite honestly, founder determination come together. And one of the many, many reasons why we're so excited to have Myogene Bio as one of CONNECT's 2026 Cool Companies and why we appreciate you joining us today.

Thanks for having me.

Thanks for listening to The CONNECT Current. If you want to learn more about CONNECT and explore resources across San Diego's innovation ecosystem, follow us on LinkedIn or visit connect.org. We'll see you next time on The CONNECT Current. Where San Diego's innovation story keeps moving forward.

This podcast was edited by Resonate Recordings.