Rare Leader: Rich Horgan, Founder and President, Cure Rare Disease
Disease focus: Broadly focused on rare genetic disorders, though starting with Duchenne muscular dystrophy before scaling to other rare genetic diseases.
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.