Toronto-based Specific Biologics Inc. 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, according to the company, which announced the financing on Monday.
The company is developing treatments for dozens of neurological and neuromuscular conditions grouped as repeat expansion disorders, which share a common genetic fault. ALS, the fatal illness also known as Lou Gehrig's disease, is its first target. The Globe and Mail reports that about 1,000 people in Canada are diagnosed with ALS each year.
Why it matters
Repeat expansion disorders arise when short sequences of DNA repeat far beyond their normal length, producing strands that damage cells. Correcting this kind of fault takes more than one cut, which puts it beyond the reach of standard gene-editing tools. Specific's approach, called Dualase, is designed to make two cuts at once and remove the excess sequence.
If the therapy performs in people as it has in laboratory cell samples and mice, the company believes it could stop ALS from progressing in patients whose condition stems from the defect. Brent Stead, Specific's chief executive, said the longer-term aim is to treat people early enough that they never develop symptoms, according to The Globe and Mail.
The financing also stands out for its ownership structure. Canadians retain a large majority of the company, including early backers Lumira Ventures and adMare BioInnovations, along with Genesys Capital, BDC Life Sciences Venture Fund and Seido Capital. Nikhil Thatte, a partner at Lumira, told The Globe and Mail that such a level of domestic ownership is unusual at this stage for a promising Canadian biotech firm.
Two cuts instead of one
The science behind Specific comes from David Edgell, a biochemistry professor at Western University in London, Ont. The Globe and Mail reports that Dr. Edgell, who grew up in Victoria fascinated by science fiction, began developing his gene-editing technique in the 2000s. He moved to Western in 2003 and turned his attention to gene editing after earlier research on stretches of DNA that appeared to have no function.
Gene editing itself is not new. The field was reshaped in 2012 when Jennifer Doudna and Emmanuelle Charpentier demonstrated CRISPR/Cas9, work that later earned a Nobel Prize in chemistry. CRISPR acts like molecular scissors, cutting a single mutated gene so the body's cells can repair it. It proved faster, simpler and cheaper than earlier methods and has produced clinical results, including Casgevy, a sickle-cell treatment approved for sale in Canada in 2024.
But single-cut editing has limits. Treatments can act on the wrong part of the DNA, an "off-target" effect that can cause serious harm, and that risk has made investors cautious. Mr. Thatte told The Globe and Mail that gene editing is "virtually unfundable in the U.S." at present, while adding that Specific has met every scientific milestone it has set.
Specific's answer is to encode what amounts to two pairs of scissors into a single protein. The Globe and Mail reports that the company packages the tool inside the shell of a harmless virus, which is injected into the spine and travels to the brain to reach the neuron cells where the fault lies. The therapy is programmed to attach at the point where the problematic repeat begins and at the far end of the runaway sequence. It cuts both ends, lifts out the faulty section and joins the remaining DNA back together. Dr. Edgell described the process to The Globe and Mail as working "like molecular Lego," leaving healthy DNA with normal repeat lengths behind.
According to the company, the treatment would require a single injection and has shown no off-target effects in preclinical work on human cell samples and mice. Specific hopes to begin its first human studies in ALS patients in 2028. Ben Scruggs, a partner at Hatteras, said there is a clear and affordable route to showing whether the platform can work.
A board drawn from Canadian biotech
Specific has assembled advisers and executives with long records in the sector. Its board includes Paul Hastings, former chief executive of the B.C. biotech pioneer QLT Inc., and serial entrepreneur Richard Glickman. The company has also brought Canadian gene-editing veterans Brad Geddes and Paul Alloway, both based in the United States, into its senior management team.
Mr. Hastings told The Globe and Mail that the technology could change how these disorders are treated, and that the payoff would be considerable if it succeeds in ALS. Mr. Stead, who trained in the same Western lab as Dr. Edgell before persuading him to let the company commercialize the research, said Specific is also in talks with a large pharmaceutical company about a development partnership.
Beyond ALS, the company sees potential for Dualase in other repeat expansion conditions, including a form of Huntington's disease, and more widely in genetic diseases such as cystic fibrosis, according to The Globe and Mail.
Watch for Specific's planned first-in-human ALS study, targeted for 2028, and for the outcome of its talks with a pharmaceutical partner.
