I spoke with Rich Horgan, founder of Cure Rare Disease, a nonprofit biotechnology company disrupting the paradigm of drug development for rare genetic diseases. Through collaborations across leading institutions, Cure Rare Disease develops customized therapies in record time.
Rich’s brother, Terry, was born with Duchenne muscular dystrophy. The disease had no treatment and is ultimately fatal. His brother’s battle with the disease was very formative for Rich and shaped his ambitions at Harvard Business School. When Rich started at business school in 2016, he asked himself: what can I do to change the future for Terry and others?
At first, Rich considered taking on a well paying job, saving up, and donating to organizations doing research in the field, but he realized that this process would take too long and not give him enough time to save Terry.
HBS’s entrepreneurial ecosystem allowed Rich to dream of a different, better future. He met with researchers, clinicians, and regulatory experts to fully and deeply understand all the work that was being done around Terry’s disease and others like it. He looked into everything from the ongoing research to the drug pipeline to in progress clinical trials and found nothing that would help his brother.
Rich had often heard of nonprofit organizations discussing how close they were to a cure but years and even decades would go by without any true news around an actual cure. These situations felt like a frustrating bait and switch, and Rich wanted to find a better way.
While in business school, Rich took an MIT course taught by Professor Jonathan Fleming on entrepreneurship in the life sciences. Fleming became one of Rich’s close mentors, and when Rich shared his story and ambitions with Fleming, he recommended Rich speak with Tim Yu, a physician at Boston Children’s Hospital.
Tim was developing a drug for Batten disease, an ALS like disease that affects children, leading to certain death within three years. Tim had met Mila, a child with Batten disease through her mother, and he wanted to do something to help. So over the course of 12 months, he developed a drug based on her precise genetic profile, and the drug helped significantly extend her life.
Inspired by Tim’s work, Rich formed his own team with the goal to save Terry. His team consisted of his brother’s long time clinician, Yale researchers, a muscular dystrophy patient, and many others across a range of different fields. They started with a muscle biopsy and then began developing a therapeutic approach for Terry. Within 6 months, they had a proof of principle that would save his brother through providing him with a structural protein that he lacked and that led to Duchenne. Throughout the process, Rich and his team partnered with more collaborators from Charles River Laboratories to regulatory consultants.
Rich and his team met with the FDA for a pre approval meeting and have started manufacturing the drug, a CRISPR drug. Terry will be the first person in the world to receive a CRISPR drug for this type of disease. It has taken Rich and his team just 3 years to evolve this therapy from idea to bedside, an incredibly impressive milestone for drug development.
Cure Rare Disease was initially founded to raise funds for these efforts but over time through countless conversations and experiences, Rich has evolved the organization that now drives a much larger mission. Rich is building an ecosystem supported by reimbursement partners to democratize access to customized therapeutics for rare diseases.
Rare diseases affect over 30 million people in the United States, but they are not common enough for big pharmaceutical companies to take an interest yet the therapies created to tackle rare diseases are held to the same testing standards as drugs created by large pharma companies.
Beyond the physical impacts, rare diseases affect patients and their families in numerous ways. The challenge patients and their families face can lead to divorce, mental health problems, burnout, and more.
Cure Rare Disease is filling a void: the lack of drugs for progressively fatal diseases.
In 2020, Cure Rare Disease developed a neutralizing antibody initiative. 30% of people with rare diseases have preexisting antibodies that prevent them from getting gene therapies. Cure Rare Disease collaborated with research institutions and impacted patients to begin a pivotal research study last year. Looking ahead, the group is focused on creating solutions for this demographic.
Reflecting on his journey, Rich shares his advice for partnership building, storytelling, and entrepreneurship:
Work with people who are there for the right reasons. Team is ultimately everything. The people you work with will determine the success of your company. When building the Cure Rare Disease team, Rich focused on people who are truly passionate about the problem and would go above and beyond in solving it, rather than solely vetting for experience. He looked for people who were not motivated solely by monetary incentives but rather driven more so by the mission and the vision. When leading his team, Rich constantly reminds them of their shared passion and the “why” behind all that they do, which continues to inspire, energize, and align everyone.
Meet people where they are. When Rich seeks to convey more complex scientific topics to a nontechnical audience, he always leverages powerful analogies that boil the explanation down to something they relate to.
Similarly, he tailors his communication to the audience he is speaking to. For mission focused audiences, he focuses on the storytelling, the narrative arc behind his own motivation in founding Cure Rare Disease. For more technical audiences, he dives more deeply into the science and the exciting research being pioneered by the group.
Ultimately, he focuses on tapping into the empathetic side that everyone has, articulating why the story matters, what the implications are, and the big vision: how the team will leverage their learnings to move from their initial use cases to many others.
Just jump in. Instead of thinking how far you have to go before you reach your end goal, take everything one step at a time.
Immerse yourself in the ecosystem, meet as many people as possible, and ask as many questions as you can. Listen and learn first and then start building. Don’t be afraid to reach out to people and ask for things. The worst they can say is no.