What does Cure Rare Disease do?

Cure Rare Disease is a non-profit biotechnology company located in Woodbridge, Connecticut, founded in 2018. The organization focuses on developing personalized genetic therapies for rare and ultra-rare neuromuscular diseases, particularly those lacking existing treatments. Inspired by the founder Richard Horgan's brother, Terry, who had Duchenne muscular dystrophy, the company collaborates with leading researchers and institutions to create innovative solutions using gene therapy, gene editing, and antisense oligonucleotides. The company operates as a "drug development engine," emphasizing patient partnerships and community funding to streamline the development of therapies for small patient populations. Cure Rare Disease aims to enable access to life-saving genetic medicines by fostering collaborations among researchers, clinicians, and donors. Its development model prioritizes individual patients, allowing them to co-fundraise for custom therapies while advancing lead candidates through FDA submission and trials. The organization is committed to addressing the challenges of ultra-rare diseases and ensuring that therapies meet the real needs of patients and their families.