Rich Horgan has assembled a team to quickly develop a gene-editing therapy for his brother’s rare form of muscular dystrophy. Other people with rare diseases hope they are next
Rich Horgan still remembers the day his younger brother, Terry Horgan, could no longer summon the strength to walk up the steps of a movie theater. Terry was born with Duchenne muscular dystrophy, a rare genetic disease that causes progressive muscle loss. Today, at 24, he’s too old to participate in one of the many clinical trials of experimental drugs for the rare disease. His heart strength is declining, and he doesn’t sugarcoat his situation: “It sucks, but you got to keep moving.”
Lately, however, Terry has something to hope for: a personalized gene-editing therapy that could provide a genetic workaround to the particularly rare mutation that causes his disease.
Two years ago, Rich started a nonprofit to fund basic research on his brother’s disease. Before long, Rich’s ambitions grew, and in the summer of 2018, he began working on a way to treat Terry. This spring, Rich’s nonprofit, Cure Rare Disease, announced the daring goal to use CRISPR gene editing to treat Terry and other individuals with rare forms of neuromuscular disease. Until recently, Terry had never even heard of CRISPR; by this time next year he could become the first recipient of a custom-designed CRISPR therapy.