Specific Biologics raises $35M to test a repeat-cutting gene editor in ALS

Most gene editors are too big to fit in one AAV vector. Specific Biologics says its Dualase system isn't, and on 2 November the Toronto company announced an oversubscribed US$35 million Series A to take it into people with ALS.

Hatteras Venture Partners led the round. Foundational investors Lumira Ventures and adMare BioInnovations returned, and new money came from Genesys Capital, BDC's Life Sciences Venture Fund, DeepWork Capital and Seido Capital.

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What Dualase does

Repeat expansions are stretches of DNA where a short sequence is copied over and over. According to the company's release, they are implicated in more than 40 genetic disorders. Dualase is designed to cut out the disease-causing expansion while leaving the normal sequence in place.

Because it is compact, it fits within a single adeno-associated virus and can be delivered straight to the central nervous system to reach motor neurons. The platform can also use lipid nanoparticle-delivered mRNA. In patient-derived ALS cell models, the company reports a near-complete collapse of the C9ORF72 repeat expansion, and in animal models it reports correction of downstream disease hallmarks with no detectable off-target effects. It has also shown preclinical editing in several other neuromuscular repeat expansions.

What the money buys

The round is sized to carry the lead Dualase candidate to initial clinical biomarker proof-of-concept in the first cohort of an adaptive Phase I/II trial in C9ORF72-associated ALS. The release does not give a start date or mention a regulatory clearance yet. Stead says the aim is to address the disease "at its genetic source".

"Gene editing has the potential to transform the treatment of genetic diseases" Ben Scruggs, partner, Hatteras Venture Partners

Chief executive Brent Stead described the financing as a defining step towards that goal. Paul Hastings chairs the board.

Why it matters

An adaptive design that reads out on a biomarker in the first cohort is a capital-efficient way to get an early answer, and $35 million is a focused budget for it. Canadian specialist and government-backed funds make up much of the syndicate, alongside a US lead.

Our take: this is a clear, staged plan, and the next 12 to 18 months will be about execution. That means CMC, regulatory and clinical operations rather than more discovery.

What it means for hiring

A first-in-human AAV gene editing study needs regulatory affairs leaders who have handled CNS gene therapy filings. In our experience, very few people have done that, in Canada or elsewhere.
Vector manufacturing and analytical development become the critical path once a company commits to a single-AAV product. Expect demand for CMC and quality leaders with AAV experience.
Running an adaptive Phase I/II in ALS calls for clinical operations and medical leads with neuromuscular trial networks. We'd expect hires here to be people who already know the ALS centres.

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