Date: Jun 22, 2018 Author: Don Seiffert Source: bizjournals (
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While a student at Harvard Business School, Rich Horgan, 26, founded a Boston nonprofit called Terry's Foundation for Muscular Dystrophy last year, and an affiliated for-profit biotech called Myos. Last month, he was named a Blavatnik Fellow, giving him the chance to work with Harvard inventors and Harvard-affiliated hospitals on advancing both projects.
But the impetus for the nonprofit and company was largely personal: Horgan hopes to find a treatment for his brother, Terry, 23, who has a genetic mutation that causes Duchenne muscular dystrophy. It's a mutation that's rare even by rare disease standards, and Rich says no one else is working on anything that could conceivably treat him.
This Sunday, June 24, Horgan is hosting what's hoped to be an annual Duchenne educational conference at Brandeis University. The event will feature leading academics, and families will be able to ask about research and treatments. Rich Horgan recently spoke to the Business Journal about his new ventures.
How was your brother diagnosed? Terry was diagnosed with Duchenne muscular dystrophy when he was five. It's a pretty nasty disease if you're not familiar: progressive muscle weakening of both the skeletal and cardiac muscle. Muscle scarring occurs, known as fibrosis, which eventually takes over and replaces where the healthy muscle used to be. Boys become wheelchair-confined at around 9 or 10 (years old) and then lose the ability to function thereafter, and traditionally pass away between 25 and 30.
Did you and your family notice signs before he was five? Yeah, my mom and dad would notice weakness with walking, crawling, kind of traditional phenotypes -- inability to get up, a lot of falling.
Is he in a wheelchair now? Actually, that's kind of the odd thing about it, where the story gets a little interesting. He's not in a wheelchair, he's actually still walking with assistance. He splits about 50-50 in the chair and walking, so we're not sure why, to be honest.
How did you get the idea for this structure of creating both a nonprofit foundation and a for-profit biotech arm? I came to business school and I knew I wanted to do something in the (Duchenne) field. I looked around, talked to a lot of researchers. I saw a lot of action -- gene therapy, exon-skipping -- and said, 'Wow, this is not really going to help my brother.' Through those conversations, I met a researcher by the name of Lou Kunkel out of Boston Children's (in 2016) and that's kind of where the story begins. Lou had a new idea for cell therapy -- essentially, programming smart cells to go into the body and recolonize. I said, 'Wow, this sounds great.' It would be mutation-agnostic, it would be age-agnostic, and there are very few therapies out there that would be amenable to that. So I said, 'How do I help?' and he said, 'Well, you've got to raise money.'
We started the foundation on Aug. 17, 2017. The foundation really was a second thought. The first thought was, 'OK, we need to throw a gala. How do we throw a gala to raise $50,000? We'll start a foundation.' In December we raised $60,000, put that into the lab a few months ago, and we're starting to see some promising stuff coming out of there.
Our conference on June 24, we've got 25 families coming, six biotech and pharma companies, and three academic clinicians. Our second gala this October will be at the Museum of Science, and we'll aim to raise $100,000. And we started a GoFundMe to raise another $100,000.
Tell me about Myos. The for-profit arm is really there to speed commercialization. It enables us to write more grants, it's there to convene a scientific advisory board so we can have eyes on the research at a very early stage. It's really geared toward translational research. We've got the leading clinicians, the leading KOLs (key opinion leaders) as part of the group, with eyes on this research -- on the personalized research, and one other project we're looking at identifying here shortly. It's all about shortening the timeline.
But when you call it a for-profit, people naturally assume that the whole point of this is to make money as some point. That's interesting when you think about for-profit in the country. For-profit businesses are an enabling factor. It enables us to raise money down the road, which we'll be doing in a few months. It's just a vehicle. Yeah, it'll need to make money some day. But priority one is effective treatments. Priority number two is, we'll make money.
Why did you decide to take this on yourself? Myos, through this Blavatnik scholarship that I'm doing through Harvard, they give you a real solid group of advisors. My advisor that's dedicated to me is Rosanna Kapler; you might know her from Nimbus (Therapeutics). She's now helping me to think about: How do you build a proper biotech? How do you vet research and how do you build a team around that? We're at the early stages. We're at floor one. But with her 30-plus years of biotech experience we're hoping to push a lot faster than me and a couple of PhDs trying to put this together ourselves.
Did you see this as the only option for trying to find a solution for a really, really rare disease? When I looked at the research, a lot of it in Phase 1, Phase 2, pre-clinical, isn't amenable to (my brother). It's for the sweet spot of 5-10 years old. What about the boys who are 15 years old and older, who've put a decade plus time on this clock? You can't forget about them. And to me it feels like we've got to do more there.
You just started in the last year, you've raised some money, now what's the first step working toward treating the disease? It's two prongs right now. The cell therapy is under development, we're continuing to (address) problems and targets there; and personalized approach, we're doing genetic sequencing on my mother and brother, and we're also doing a muscle biopsy to identify what's going on here. Once we get that, it's on to step two: What agent can we use to effect this change?
How have you been received by other Duchenne foundations? To be honest, I don't know. What's missing, and the big takeaway from your question is, there needs to be more collaboration. We've all got too small of pots of money to be fighting one another, and that's really what's needed to get this done.
Is your brother involved in the organization? Yeah, he has two jobs. The first is to smile for pictures, which he struggles with. Job number two is he does all our IT stuff -- the website, and anything related to that. He went to Cornell undergrad for information science, and just graduated last May, so we're all proud of him.
