Specific Biologics raises $35M Series A to take ALS gene editing into the clinic
What's the deal? Toronto-based Specific BiologicsDealroom has a profile for this one. Try Dealroom → has closed an oversubscribed US$35 million Series A to advance its lead gene-editing candidate toward human trials. Hatteras Venture Partners led the round, joined by foundational investors Lumira VenturesDealroom has a profile for this one. Try Dealroom → and adMare BioInnovationsDealroom has a profile for this one. Try Dealroom →.
New backers Genesys CapitalDealroom has a profile for this one. Try Dealroom →, the Business Development Bank of Canada's Life Sciences Venture Fund, DeepWork CapitalDealroom has a profile for this one. Try Dealroom →, and Seido CapitalDealroom has a profile for this one. Try Dealroom → also participated.
What's the endgame? The company develops precision gene-editing therapies for serious genetic diseases. Its proprietary Dualase platform is designed to remove disease-causing DNA repeat expansions while preserving the normal sequences cells need to function.
Those abnormal repeats are implicated in more than 40 genetic disorders, and Specific says it has shown editing in preclinical studies across multiple neuromuscular repeat expansions.
Where the money goes: The proceeds will fund the lead Dualase candidate through initial clinical biomarker proof-of-concept in the first cohort of an adaptive Phase I/II trial. The target is C9ORF72-associated amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease with no approved therapies.
"Our goal is to address the disease at its genetic source to create a new therapeutic option for patients who have waited far too long," said chief executive officer Brent Stead.
Why now? Investors framed the raise around reaching the clinic. Lumira partner Nikhil Thatte said early clinical proof-of-concept in ALS patients "represents an important inflection point for the company."
What could go wrong? Gene editing still faces hurdles in precision, specificity, and delivery. Hatteras partner Ben Scruggs acknowledged these "longstanding challenges," and the Dualase evidence remains preclinical — the Phase I/II trial is the first real test in humans.
The signal: At $35 million, this ranks among the larger health Series A rounds in Canada, sitting in the 92nd percentile across 232 comparable deals. The round reflects continued investor appetite for gene-editing platforms targeting diseases long considered beyond reach.
Read more: prnewswire.com
Image credit: Specific Biologics
