Virica Biotech joins forces with NRC in quest to create treatment for rare genetic disorder

Gene therapy stock image
Gene therapy stock image

An Ottawa-based startup is poised to play a leading role in the National Research Council of Canada’s campaign to develop an affordable treatment for a rare and potentially deadly genetic disorder.

Virica Biotech announced this week it’s landed $400,000 in federal government funding to help the NRC manufacture a gene therapy to treat lipoprotein lipase deficiency, or LPLD.

Canada has the world’s largest population of LPLD patients, with many of them concentrated in the Saguenay region of Quebec, where someone with the genetic mutation settled hundreds of years ago. While the disorder affects about two people in a million worldwide, as many as one in 50 people carry the mutation in Saguenay.

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People with the inherited disease are unable to break down fat particles in their blood and must avoid eating fat in order to ward off recurring and potentially fatal attacks of pancreatitis. There is currently no effective treatment or cure for LPLD.

While researchers at the University of British Columbia came up with a method to fix the malfunctioning gene responsible for the disease, the Dutch manufacturer that licensed the treatment pulled it off the market several years ago after selling just one course of the drug, which cost $1 million for a one-time dose.

Now, the NRC is reviving efforts to find a cost-effective treatment for LPLD, and Ottawa’s Virica hopes to be a big part of the solution. 

Founded in 2018, the firm makes specialized compounds that help boost production of viral vectors, which deliver new genes into infected cells designed to repair defective genes responsible for diseases such as cancer, muscular dystrophy and hereditary blindness. 

“I thought this was a really great opportunity to show the value of Virica’s technology to boost viral production,” Virica co-founder and CEO Jean-Simon Diallo told Techopia on Tuesday. “We’re really excited.”   

“For us, it’s actually a really exciting proof of concept.”

Together, Virica and the NRC hope to develop an affordable version of the viral vectors that treat LPLD, known as adeno-associated viral vectors, or AAV. One of the biggest obstacles to developing effective gene therapies so far has been the high cost of manufacturing the specially engineered viruses, and Diallo hopes to help change that.

“For us, it’s actually a really exciting proof of concept,” he said. “A lot of companies are developing gene therapy products that use AAV.”

The budding collaboration with the world-renowned research facility is just the latest win for Virica, which announced earlier this year it’s inked a partnership with Oxford Biomedica to help the publicly traded British firm improve the yield and production efficiency of its next-generation gene therapies. 

That announcement came on the heels of a multimillion-dollar funding round aimed at helping the Ottawa company accelerate its research efforts. Diallo says Virica now has contracts with more than 35 manufacturers of gene therapies that are looking to his startup to help make their treatments cheaper and more effective. 

The firm’s revenues have been rising at a steady clip over the past two years, and Virica’s headcount has jumped from 16 to nearly 30 in the past eight months as demand for its technology surges. 

Meanwhile, the company is already on the verge of outgrowing its new 3,000-square-foot home at the University of Ottawa Heart Institute that just opened last October.

“We’re off to the races now,” Diallo said. “The reality is there’s a huge demand for what we do. There’s a lot of promise on the horizon.”

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