Rich Horgan spearheaded a gene therapy for his brother. The trial ended in tragedy, but the work continues for more patients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

In his first in-depth interview since the death, Horgan told Endpoints News the nonprofit is continuing work on more than 15 additional therapies in its pipeline aimed at ultra-rare diseases. First, the group must overcome a dosing issue that was cited as the likely cause of Terry’s death.

“Terry is not coming back. And I miss him every single day of the week. But the best I can do for Terry with the time that I have left is to try to advance this model for other ultra-rare patients,” Horgan said.

But given Terry’s death, an...