Specific Biologics has closed an oversubscribed US$35 million Series A to take Dualase, its two-site gene editor, into a first human trial for C9ORF72-associated ALS — the most common known inherited form of the disease. Hatteras Venture Partners led the 2 November 2026 round, joined by foundational investors Lumira Ventures and adMare BioInnovations, both of which backed the Toronto company's earlier seed financing.
The round in four figures
Figures from Specific Biologics' own announcements and public grant records.
A repeat sequence, cut down to size
C9ORF72-associated ALS traces to a single, expanded DNA sequence: a repeating six-letter motif in the C9ORF72 gene that, past a threshold length, produces toxic RNA and protein and is the most common known inherited cause of ALS. Specific Biologics, a Toronto company spun out of Western University research in 2017, is developing Dualase to cut that expansion down rather than work around it.
According to the company, Dualase's lead candidate achieved near-complete collapse of the pathogenic C9ORF72 repeat expansion in patient-derived ALS cell models, with similar effects reported in animal models and no detectable off-target effects. Those results are the company's own preclinical data, not yet replicated by an outside lab or tested in a person; the Series A funds the first attempt to do the latter.
“This financing represents a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS.” Brent Stead, Chief Executive Officer, Specific Biologics — 2 November 2026 announcement
Two cuts, where gene editors usually make one
Most gene-editing nucleases cut DNA at a single site. Dualase is built around a two-site, dual-cleaving mechanism that the company says generates non-compatible ends and is engineered to exploit the cell's own DNA repair pathways — intended to make the removal, repair or insertion of DNA sequences more precise than a single cut allows. The platform is designed to be delivered either by a single adeno-associated virus (AAV) vector or by lipid-nanoparticle-delivered mRNA, aiming at a limited-dose treatment rather than a chronic one.
Three things Dualase is betting the round on
Two cuts, not one
A dual-cleaving, two-site mechanism designed to generate non-compatible ends and exploit the cell's own repair pathways, aiming for more precise edits than a single-cut nuclease.
Built to erase the repeat
Preclinical data the company describes show near-complete collapse of the pathogenic C9ORF72 repeat expansion in patient-derived ALS cells and similar effects in animal models — not yet shown in a person.
One dose, not a chronic one
Delivery by a single AAV vector or lipid-nanoparticle mRNA points toward a limited-dose treatment, unlike the repeat-dosing antisense approaches most of the C9ORF72 field has tried.
A category still mostly selling tools, not medicines
Gene-editing market forecasts vary enormously depending on what they count. Broad “genome editing” figures — which bundle research tools, reagents and services in with actual treatments — run into the tens of billions of dollars by 2030. A narrower estimate for gene-editing therapeutics alone, from the same research firm, is a fraction of that. The gap says less about any single company's prospects than about how early the therapeutics side of the category still is.
A thirty-fold gap in the same category
$1.0B → $30.23B
BCC Research's narrower 2029 forecast for gene-editing therapeutics alone, against two broader 2030 forecasts for the full genome-editing category (tools, reagents and services included) from BCC Research and Reports and Data.
Sources: BCC Research, Gene Editing Therapeutics Market · BCC Research, Genome Editing Market to Grow at 16.9% CAGR Through 2030 (via GlobeNewswire) · Reports and Data, Genome Editing Market Size (via BioSpace)
Regulatory News outlook
We read the gap between the narrow and broad forecasts as a sign that gene-editing therapeutics are still a small fraction of a market mostly selling research tools, not approved medicines — and that a disease like C9ORF72 ALS, with no approved gene-editing treatment yet, is exactly where that could start to change.
How we got here: BCC Research's own $1.0 billion 2029 figure for therapeutics alone against its $23.7 billion 2030 figure for the full genome-editing category implies therapeutics are well under 5% of the category today, even before adjusting for the extra year between the two forecasts.
Dualase against C9ORF72's other approaches
| Program | Modality | Delivery | Stage |
|---|---|---|---|
| Dualase (Specific Biologics) | Two-site gene editor | Single AAV vector or LNP-delivered mRNA | Preclinical; Phase I/II planned |
| BIIB078 (Biogen/Ionis) | Antisense oligonucleotide | Intrathecal injection | Discontinued after Phase 1 (missed efficacy) |
| WVE-004 (Wave Life Sciences) | Stereopure antisense oligonucleotide | Intrathecal injection | Phase 1b/2a (FOCUS-C9, initiated 2021) |
| Qalsody / tofersen (Biogen) | Antisense oligonucleotide | Intrathecal injection | FDA-approved 2023, for SOD1-ALS, a different genetic subtype |
From company and publisher announcements and public ALS pipeline reporting; not a clinical comparison. Qalsody targets SOD1-ALS, a different genetic subtype than C9ORF72-ALS, and is included for approval-pathway context only.
From a Western lab to a Series A
Specific Biologics' road to a first human trial
- 2017
Specific Biologics founded
Spun out of gene-editing research at Western University in London, Ontario; the company later establishes headquarters in Toronto.
- December 2021
Seed financing closed
Lumira Ventures and adMare BioInnovations back the company as foundational investors; the Cystic Fibrosis Foundation separately awards a Therapeutic Development Award for Dualase.
- March 2026
Genome Canada grant
A CAD$1.8 million award with Western University funds a machine-learning platform to accelerate Dualase editor design.
- 2 November 2026
$35 million Series A announced
Hatteras Venture Partners leads an oversubscribed round, joined by Lumira Ventures and adMare BioInnovations.
- Ahead
Phase I/II biomarker proof-of-concept
The round funds Dualase's first clinical trial, an initial biomarker proof-of-concept study in C9ORF72-associated ALS; no start date disclosed.
What the first human dose still has to answer
Every claim Specific Biologics has made about Dualase's precision and effect on the C9ORF72 repeat expansion comes from cell and animal models, described by the company rather than published in a peer-reviewed paper Regulatory News could independently review. That is ordinary for a company at this stage, and worth stating plainly: the Phase I/II trial the Series A funds is Dualase's first test in a human being, in a disease where one prior antisense program was discontinued for missing efficacy and another remains in early-phase trials of its own.
- Phase I/II trial: an initial clinical biomarker proof-of-concept study in C9ORF72-associated ALS; no registered trial, enrollment target or start date disclosed.
- Delivery route: the company has described both single-AAV and lipid-nanoparticle mRNA delivery options; which one enters the clinic first has not been specified.
- Board: the round follows a December 2025 appointment of biotechnology leader Dr. Steven Kanner to Specific Biologics' board, announced alongside an earlier additional investment.
- Pipeline beyond ALS: the company has also described Dualase programs in other repeat-expansion and genetic disorders, including earlier grant-funded work on cystic fibrosis nonsense mutations; none has a disclosed timeline.
What is not yet public: Dualase's specific preclinical dataset in a peer-reviewed form, the Phase I/II trial's registration details, and the full investor list beyond Hatteras Venture Partners, Lumira Ventures and adMare BioInnovations. Regulatory News will update this page as those details are confirmed.
Sources & further reading
- Specific Biologics, “Specific Biologics Announces Oversubscribed US$35 Million Series A Financing to Advance Gene-Editing Therapy for ALS,” press release, 2 November 2026. ca.finance.yahoo.com
- adMare BioInnovations, “Specific Biologics Announces Oversubscribed US$35 Million Series A Financing to Advance Gene-Editing Therapy for ALS.” admarebio.com
- Dealroom, “Specific Biologics raises $35M Series A to take ALS gene editing into the clinic.” dealroom.co
- FinSMEs, “Specific Biologics Raises $35M in Series A Funding.” finsmes.com
- Schulich School of Medicine & Dentistry, Western University, “New funding advances precision gene-editing technology.” schulich.uwo.ca
- pharmaphorum, “New blow for ALS pipeline as Biogen and Ionis candidate fails Phase 1 trial.” pharmaphorum.com
- BCC Research, Gene Editing Therapeutics Market. bccresearch.com
Regulatory News reports on public regulatory documents. It is not legal advice, and the primary sources above govern. If we have made an error, we will say so in public: see corrections.
Frequently asked questions
What did Specific Biologics announce?
An oversubscribed US$35 million Series A financing, announced 2 November 2026 and led by Hatteras Venture Partners, joined by foundational investors Lumira Ventures and adMare BioInnovations. The funds advance the company's lead Dualase candidate into a Phase I/II trial for C9ORF72-associated ALS.
What is Dualase?
A gene-editing platform that cuts DNA at two sites rather than one, designed to remove disease-causing DNA repeat expansions while minimizing unintended edits. It can be delivered by a single AAV vector or by lipid-nanoparticle-delivered mRNA, according to the company.
Has Dualase been tested in people?
No. All reported results are preclinical, from patient-derived ALS cell models and animal models. The Series A funds the company's first Phase I/II clinical trial, an initial biomarker proof-of-concept study in C9ORF72-associated ALS.
What is C9ORF72-associated ALS?
The most common known inherited form of amyotrophic lateral sclerosis, caused by an expanded, repeating DNA sequence in the C9ORF72 gene. Dualase is designed to shorten that repeat expansion at the DNA level.
