Brotherly love
Hub entrepreneur trying to cure sibling’s rare muscle disease
Rich Horgan, 28, used to hold his breath every time his mom called — he was worried about his younger brother, Terry, who has Duchenne muscular dystrophy, which caused frequent falls that slowly cut down his chances of ever walking again.
So Horgan decided not to wait around for a cure for his brother — he decided to put a team together to create his own cure, using Terry’s cells.
“There’s nothing else out there to try or even really hope for so you’re kind of in a position where you really have no choice,” said Terry, 26.
Horgan founded the Boston-based biotech nonprofit Cure Rare Disease about a year and a half ago, a whirlwind period of time during which he assembled a team of scientists set on the urgent mission to cure Terry and others with Duchenne’s.
Duchenne muscular dystrophy is a rare disease that had already impacted three of Rich and Terry’s uncles, and their mom is a carrier.
Patients who have it used to barely make it to their teens, but now can live into their thirties or beyond.
Given that time crunch, the Horgans got to work right away on collaborating with researchers. In the spring of last year, Horgan got news that Terry’s cells had been fully corrected in a petri dish.
“We have tried to break down the walls and silos that are typical for drug development,” said Horgan.
The nonprofit is implementing a groundbreaking methodology with precision medicine by developing customized therapeutics designed using
CRISPR gene-editing technology in collaboration with researchers at Charles River Labs in Wilmington.
Terry said, “There’s nothing out there right now or even in the pipeline other than this. Without this there is no hope.”
Duchenne’s causes progressive muscle weakness, which also impacts the heart, and is only found in 1 of 3,500 boys worldwide. It is rarely found in girls.
Lauren Black, a distinguished scientist at Charles River Labs, heard of Terry’s “dire and compelling” story and jumped on board to assist with research.
“When you know you have something that someone else needs badly, it’s an immediate yes,” said Black.
Black is now growing rat models with Duchenne’s to supply to doctors who can test the drugs on the rats.
“You know what is broken in Terry, there is a gene and it needs to be fixed,” said Black.
Horgan said they’re paving the way for the application of the world’s first human administration for Terry, expected in late 2020 pending FDA approval.
“It’s quite scary to be the first one because the knowledge about the drug is limited,” said Horgan.
“It’s pretty groundbreaking, he does all the science and I show up wherever he tells me to,” joked Terry.
Horgan and Terry, along with the team at CRD, are continuing a race toward a cure with hopes set on curing other rare diseases as well.
“The existing ecosystem isn’t entirely conducive to rare disease so that’s one thing we are trying to modify,” said Horgan.