Specific Biologics Inc., a Toronto biotechnology company, said Monday it has closed a US$35-million venture capital round to advance a gene-editing therapy aimed at slowing or halting ALS in the roughly one in 10 patients whose disease is caused by a specific genetic defect, The Globe and Mail reports.
The round was led by North Carolina-based Hatteras Venture Partners. Canadian investors, including early backers Lumira Ventures and adMare BioInnovations, along with Genesys Capital, BDC Life Sciences Venture Fund and Seido Capital, still hold a large majority of the company — an ownership split that Lumira partner Nikhil Thatte told the Globe is uncommon for a promising Canadian biotech firm at this stage.
The financing supports a technology called Dualase, developed over two decades by David Edgell, a biochemistry professor at Western University in London, Ont. Specific is initially targeting amyotrophic lateral sclerosis, the fatal condition also known as Lou Gehrig's disease. About 1,000 people are diagnosed with ALS in Canada each year, according to the Globe.
Why it matters
ALS belongs to a family of conditions called repeat expansion disorders, in which short stretches of DNA repeat far beyond their normal length and produce toxic effects. Specific believes its approach could address dozens of neurological and neuromuscular diseases that share this kind of defect, and the company says the same platform could later be turned to other conditions, including a variant of Huntington's disease and, more broadly, genetic diseases such as cystic fibrosis, according to the Globe.
The company's chief executive, Brent Stead, told the Globe that the long-term aim is to reach patients early enough "so they never become symptomatic." He studied in the same Western lab as Dr. Edgell and later persuaded him to let the company commercialize the research.
Specific has assembled a board and management team drawn from across the Canadian biotech sector. Its board includes Paul Hastings, former chief executive of B.C. biotech pioneer QLT Inc., and serial entrepreneur Richard Glickman. The company has also brought U.S.-based Canadian gene-editing veterans Brad Geddes and Paul Alloway into senior management. Hastings told the Globe the technology could change how these disorders are treated, adding that the payoff would be significant if it works.
Two cuts instead of one
Most gene-editing tools, including the CRISPR/Cas9 system that Jennifer Doudna and Emmanuelle Charpentier demonstrated in 2012 and later won a Nobel Prize for, are designed to make a single targeted change in DNA. That approach has produced clinical results — Health Canada in 2024 approved the sale of Casgevy, a sickle-cell treatment developed by Vertex Pharmaceuticals and CRISPR Therapeutics — but it also carries risks. Editing can hit the wrong part of the genome, producing what the field calls off-target effects, and Thatte told the Globe that gene editing is "virtually unfundable in the U.S." at present. He added that Specific has met each of its scientific milestones so far.
Dualase is built for a problem a single cut cannot solve. In repeat expansion disorders, the repeated sequence grows longer and longer, and the fix requires two edits. Specific's design encodes what amounts to two pairs of molecular scissors into a single protein. The therapy is packaged inside the shell of a harmless virus, injected into the spine, and carried to the brain, where it targets neurons.
Once in place, the treatment is programmed to bind at the point where the faulty repeat begins and at the far end of the runaway sequence. It cuts both ends, lifts out the problematic stretch, and joins the remaining DNA back together — a process Dr. Edgell compared to molecular Lego, leaving healthy DNA with normal repeat sequences behind. The company says the approach requires a single injection and has shown no off-target effects in preclinical work on human cell samples and mice.
Dr. Edgell's path to the work began in Victoria, where he grew up reading science fiction and watching Star Trek. He enrolled in kinesiology at the University of Victoria, found it insufficiently scientific, and switched to molecular biology. After joining Western in 2003, he focused on gene editing, having earlier studied stretches of DNA once dismissed as "junk." His childhood interest in Lego, the Globe notes, turned out to be a fitting preview of the cut-and-rejoin mechanism at the heart of Dualase.
What to watch
Specific hopes to begin its first human studies in ALS patients in 2028. Ben Scruggs, a partner at Hatteras, told the Globe there is a clear and cost-effective route to showing whether the platform can work. Stead also said the company is in talks with a large pharmaceutical company about a development partnership.
