An Ottawa biotech startup that says it’s close to developing a breakthrough treatment for a deadly form of muscular dystrophy has received a US$1 million injection of new equity to accelerate its research efforts.
Founded three years ago, Satellos Bioscience is based at the Ottawa Hospital Research Institute. Scientists at the 10-person firm are working on a drug that will help regenerate muscle tissue in patients who suffer from Duchenne muscular dystrophy, a muscle-wasting disease that primarily afflicts boys.
Parent Project Muscular Dystrophy, a U.S.-based non-profit organization that funds research into the disease, announced the seven-figure investment this week. The group lauded the Ottawa startup’s treatment, which it says is showing promising results in laboratory testing on mice.
“This investment from PPMD will enable the Satellos team to build on their proof of concept by amplifying their efforts to invent and refine a drug development candidate suitable for testing in humans,” it said in a statement.
Duchenne is the most common type of muscular dystrophy, affecting about one in 5,000 people.
"Duchenne muscular dystrophy is a lethal disease."
Those who suffer from the genetic disorder have a severely compromised ability to generate cells that repair injured muscles. Over time, most patients are confined to wheelchairs, and many eventually require ventilators to help them breathe. Few people with the disease survive past their mid-30s.
“Duchenne muscular dystrophy is a lethal disease,” said Michael Rudnicki, a senior scientist at the Ottawa Hospital Research Institute who co-founded Satellos and serves as its chief scientific officer.
Rudnicki and his team of four Ottawa researchers say they’ve found a way to slow muscle decay in Duchenne patients using a novel drug that mobilizes the body’s own muscle stem cells to repair and regenerate muscles.
“We’re targeting the stem cell with drugs, kicking it into gear to promote repair,” he explained.
Satellos has already received about $5 million in seed funding from Bloom Burton, a Toronto-based investment firm that focuses on companies in the health-tech sector. Rudnicki said the latest capital will allow his venture to continue testing different variations of its drug in the hope of finding the one that’s most effective.
Effective treatment 'very close'
“We’re within a stone’s throw of a (drug) development candidate,” he said. “We’re very close.”
Rudnicki said the startup hopes to launch clinical trials in humans within 12 months, but that will depend on whether it can secure additional funding. He said he’s confident the firm will find more partners to help finance its efforts later this year.
“We’ve attracted a lot of interest, but the big investors want to wait and see,” Rudnicki added.
The University of Ottawa molecular biology grad said that although the new research is promising, it won’t provide a cure for Duchenne.
Rather, he said, the treatment aims to slow the progression of the disease and help patients live better, longer lives.
“A huge advance for these patients would be to be able to use a remote control, a keyboard or a mouse as they get older,” Rudnicki said. “In theory, if this works extremely well, we could see this going from being a lethal disease to being a chronic disease.”