Rare Leader: Rich Horgan, Founder and President, Cure Rare Disease

The Basics
Name: Rich Horgan

Title: Founder and president

Organization: Cure Rare Disease

Social Media Links:

Disease focus: Broadly focused on rare genetic disorders, though starting with Duchenne muscular dystrophy before scaling to other rare genetic diseases.

Headquarters: Boston

How did you become involved in rare disease: I have a younger brother Terry who suffers from Duchenne muscular dystrophy and he’s 24 years old. The disease itself goes back even further than just my brother and, unfortunately, took the lives of my mother’s three brothers in the 1970s and 1980s. And, with the advancement in the life sciences, I feel as though Duchenne and a lot of these rare diseases have been neglected, especially for patients with very rare mutations within an already rare disease. With the opportunity to attend Harvard Business School, I built the network to try and strike back at the disease. At the same time, one of the people I met was Tim Yu at Boston Children’s Hospital, who developed a customized therapeutic for a little girl with Batten disease. While his story was unfolding, I was building the team that would pick up and move forward with other rare diseases that weren’t CNS focused through the development and implementation of a stellar collaboration that includes six research institutions and the top luminaries and up and coming talent in the field. Combining these folks together gave us the platform off which we could move forward to develop the first custom therapeutic for Duchenne muscular dystrophy.

Previous career: Business development in technology sector and started a car wash with my family after I graduated college.

Education: B.S. in applied economics and management from Cornell University; MBA from Harvard Business School

The Organization
Organization’s mission: Our mission is to bridge the gap between researchers and a cure for a lot of these rare and ultrarare diseases. Ultimately, what we’re trying to do is develop customized therapeutics to treat rare disease patients in time so that today’s patients can benefit from tomorrow’s cures.

Organization’s strategy: The strategy is to prove that, first, this isn’t a crazy idea and we can do it. That’s what we’re proving with our first patient, who’s my brother. We’ve also got three other patients with potential cures for them under development, but at an earlier stage. Our hope is that we can prove the concept of customized therapeutics for what’s arguably one of the hardest diseases to treat—that being muscles since they are 40 percent of your body. Our belief is that if we can prove the concept in progressed patients in a very difficult disease, we can take the learnings from that and then apply it to other diseases and other mutations, many of which pharma will never focus on because they are either rare or ultrarare. I’m talking about mutations that are not covered by big pharma like a gene therapy approach. The cool part of what we’re doing is we’re using previous learnings to try and short circuit and decrease the time it takes to customize the therapeutics based on previous learnings and successes.

Funding strategy: I would say we have a three-tiered fundraising strategy. The first is major donors—connecting with a wealthy individual who really understands and sees the vision for a better tomorrow driven by customized therapeutics. One could argue that there is no better drug than the one that’s designed for that person. We’ve received major donor gifts from some pretty well-known global philanthropists. We do a lot with corporate partnerships. In August we had a corporate partnership with Global Partners. We were in 300 convenience stores with a donation box and the employees doing a dollar-ask campaign. We raised like $65,000 in 30 days. We’ve since expanded those partnerships. The third bucket is events. We hold a gala golf tournament and gala event. We’re looking to add additional events that are unique in nature to exemplify who we are.  

What’s changing at your organization in the next year: I would say 2020 is going to be a big one for us. The reason is that that’s when we’re expecting to dose the first patient, my brother. That’s really going to be a significant value inflection point for Cure Rare Disease, and I would argue rare disease as a whole because we’ll prove the potential in a later stage patient. It will be the first Crispr in vivo application. Hopefully, we have good results there. We’ll continue to build up the fundraising capacity to support more of these customized therapies. We’re engaging with payers to look at developing a new framework for reimbursement for these that will enable the long-term success of this program. More money, more board members, payers, dosing our first patients. I don’t think I missed anything.

Management Style
Management philosophy: I would say my management philosophy is aligning incentives in the best way possible. By that I mean getting people who are impacted by rare disease personally, people who have a stake in the game, people who are driven, people who want to see a change in the world, and basically pulling those people together and then figuring out how we can work most effectively together to build this organization rapidly. I’m convinced we could have a $5 million or $10 million organization five or ten years from now, but we just don’t have the time. One of the overarching themes of Cure Rare Disease next to transparency and collaboration is urgency. These guys touched by fatal rare diseases have a very short window of opportunity to get treated. What I hope is that we’re able to build and grow the organization fast enough so that we can have today’s patients see tomorrow’s cures.

Guiding principles for running an effective organization: I would say radical transparency is one of them. I would say passion is another one. You have got to be the hardest working people on the block. If you’re not, there will be somebody else who is willing to be harder working than you. You need to look for collaboration. A lot of our success has been driven by partnerships that we’ve made, people who we’ve convinced that customized therapies is the way to go. That’s been one of our driving factors—this ability to collaborate and partner with organizations to deliver win-wins for both parties. I often feel as though with nonprofits, giving money seems like giving money to a black hole. What’s different about us is that we have tangible goals, tangible milestones, and a very tangible endgame in mind: dosing the patient with a customized therapy is the angle for every single individual patient that comes into this organization. With us, a donor can see where their money goes. We have 90, 95 plus percent of the capital going to research. I don’t take a salary from this. What that motivates is effective outcomes in pushing things forward.

Best way to keep your organization relevant: The organization is driven by the frustration that a lot of rare disease families feel. Myself personally and many others that are either on the board, or families that I’ve had the chance to connect with through this journey, are all united by the theme of being told by a doctor to go home and leave it to the professional. Well, the professionals unfortunately just haven’t made enough progress so far. I believe that it is up to the patients to lead the charge against rare disease and that it is happening. A lay person who is not as involved in rare disease may wonder is this even possible? How can this organization do it if a large pharmaceutical company, armed with billions of dollars, has been unable to so far. What we try to do is convey the message that we’re designing therapies based on the individual that we’re expecting to cost $2 million or less. In the long-term, they will cost $1 million or less. It is possible. Look at Tim Yu [Children’s Hospital Boston]. He’s been a successful case where this has worked well. Yes, there are differences, but there are relevant similarities between what he did and what we’re doing. In fact, he’s an advisor of ours. I think that message resonates with people. I think people are tired of the lack of progress, tired of not pushing forward, and tired of losing their loved ones. That keeps it very relevant.

Why do people like working with you: I think people are attracted to passion. I think people want to hope that there can be a better tomorrow. I think what’s unfortunate about a lot of incumbent organizations is that they sort of promise a cure is close for years and years and years. But unfortunately, delivery has fallen short. What we’ve been able to do in less than a year is design a customized therapeutic that cured the individual’s cells in a dish. That was in less than a year. In the next year, keeping it under two years from bench side to bedside, we are planning to dose the patient after going through the proper preclinical testing and getting the blessing of the FDA. I think people are very attracted to that and say, “Well, if we are able to help support this organization, could we expand its capacity to help others who are touched by rare disease?” And the simple answer is, “Yes.” What we want to do is have more and more patients that we’re putting through this process and build a scalable process, and not one that is just the same thing every time. People can help effect that change. A mom in Wisconsin hosting a bake sale, or a fortune 100 company engaging in a partnership with us, all make a big difference. It’s not this black box where money goes in that nobody has any idea where it went. We did this campaign with global partners and their money went to support and grow our mouse colony at Charles River Labs. It’s super transparent. It’s super trackable. I think that’s what’s been missing in science: A) collaborations and breaking down silos, and B) this radical innovation and transparency to propel things much faster.

Mentor: I’ve got several mentors. Jamie Heywood is a big mentor of mine over at PatientsLikeMe. Brad Margus is another mentor of mine, who’s had loved ones impacted by a very rare disease. Folks like Lou Kunkel—a luminary in the field of neuromuscular diseases. He discovered and cloned the dystrophin gene in 1986 and has really been a voice of authority. He was where I started my journey about two years ago. One night I was down and out with what was going on with my brother and feeling very powerless and hopeless. I started emailing researchers and luminaries in the field and said, I’m a 25-year-old guy. I have a brother with Duchenne. Can we talk? Stephanie and Jessie Herzog are great folks who have been affected by Duchenne. They have a younger son Max with it who is four. They’re really a voice that I know I can really count on for guidance or help or support. My mentors do run the gamut.

On the Job
What inspires you: At the end of the day what inspires me is my brother. The whole reason that this organization exists and that we’ve pushed science to new boundaries is because at three years old, Terry was diagnosed with a fatal disease. Although I was only seven at the time and not really conscious of the implications of that, growing up with him and seeing him struggle first to run, then to walk, then to even do every day independent activities like get up from the chair, or sit down, or any number of things that we all take for granted, that made a big impact on my childhood and the results of that was at first a feeling of powerlessness, hopelessness, and anger at this unseen threat that was killing my brother. That encouraged me to do the best I could to try and get an opportunity to fight back. Doing well in school, going to Cornell and graduating top of the class, going to Harvard Business School all flowed into having an opportunity to strike back, which is where we are now. At the end of the day, Terry and my family are my inspiration.  Beyond that are these families with kids who suffer from diseases that they’ve never even heard of but are nonetheless thrust into this fight. And a desire to want to do things differently moving forward into the future that’s not necessarily driven by a profit motive.

What makes you hopeful: The team, the board, my little army that I’ve managed to build here to fight this disease that’s claimed tens of thousands of people throughout history. The scientists, clinicians, and researchers who work day and night to advance the field and advance our therapeutic platforms.

Best organization decision: I’m going to give you two. The first is really bringing people into the organization whose incentives are aligned with our mission. It sounds obvious, but it’s much harder to execute in real life because these people will work tirelessly to advance the cause and buy into the vision that we can have a better tomorrow. The second thing is this open collaboration that we have with our six institutions. These guys all knew of each other in the field, but many of them had never actually worked together. How can you be the best and brightest in the field and yet have never worked together? That’s because incentive mechanisms that exist don’t encourage collaboration. By breaking down those barriers and saying, “You collaborate. We’ll support your work,” we’ve been able to reach new boundaries in very short periods of time.

Hardest lesson learned: I would say the hardest lesson that I’ve had to learn is that big organizations that you think are doing transformative and groundbreaking work oftentimes are just talking a good game. It’s really the nimble, lean, and passionate organizations who are bringing all the change in this world. To me, that was not at all obvious. When you see fill-in-the-blank, big organization, you think it just spits out innovation. What you don’t see, or at least what I didn’t see, was the bureaucracy that’s involved in a large organization. They are bureaucratic, which means they oftentimes move slower, which makes them make decisions slower, which ultimately means that things they touch that should take a day or two are taking weeks or a month. It’s up to us when we work with big organizations to break through those bottlenecks and break through by saying these are patients alive here that matter at the end of the day. We’re not looking to get rich off this. We’re looking to save sick kids.

Toughest organization decision: The toughest decision we’ve had to make is choosing our collaborators. We had to choose people who were willing to work together. We were fortunate that they were the best in the field. Choosing the wrong collaborators would have spoiled things from the beginning and halted progress.

Biggest missed opportunity: I would love to have had more patients in our first cohort. We need to raise funding faster. At the end of the day, in addition to getting this reimbursed by insurance, I want to get the process approved by the FDA. Having top FDA advisers, who are familiar with the FDA and have gone through the process and been involved with decision-making at the FDA. We understand that this process needs to be to be approved by the FDA and needs to be applied to a larger cohort of patients using the same process and measuring the outcomes by standardized endpoints. I want to do that. That’s really one of the visions, next to getting this reimbursed by payers, to get the process approved so that we can move even faster.

Like best about the job: I like that I get to come into contact with so many people doing such amazing things who I can learn from and offer my perspective on things that have worked and things that haven’t worked. We have a goal in mind and a vision that’s completely doable that’ll really add value to the world and help people in ways that they haven’t been able to be helped before. The chance to work with an impassioned group of people who truly believe that the world can change. At the end of the day, it all does come down to the people and the people are one of the greatest things that I like about this job.

Like least about the job: It’s unfortunate and something that people don’t realize that often nonprofits are assumed to work together. But when you get down to the nitty-gritty of it, I think oftentimes organizations are very hesitant to collaborate with other organizations, not just with us, but in general. That’s unfortunate because a lot of times egos get in the way and say, “Well, if I can’t win that, neither can you.” People just don’t believe that happens, but it happens every day of the week. That, by far, is my least favorite part of the job—the egos of people who could help enable a better future, but because they’re not leading the charge, they don’t want to.

Pet peeve: People who don’t believe the future can be better and don’t have the courage to dream there could be a better tomorrow.

First choice for a new career: I like biotech. I want to stay here. In terms of idealized career, I want to work with people as passionate as myself and who are as into change that could be, and who want to drive that vision into reality.

Personal Taste
Most influential book: Zero to One: Notes on Startups, or How to Build the Future by Peter Thiel, Bake Masters, et al.

Favorite movie: I’m a Star Wars guy

Favorite music: I’m on the Taylor Swift bandwagon, but my formal answer would be the Foo Fighters

Favorite food: Sushi and anything that sort of swims in the ocean, except tilapia

Guilty pleasure: I have the worst sweet tooth in the world. Mike’s Pastry in the north end of Boston is a weakness of mine.

Favorite way to spend free time: With my family, my dog, and my friends. My mind is always on, and that’s really tiring. I like opportunities that force you to disconnect.