Specific Biologics closed a US$35 million Series A financing led by Hatteras Venture Partners, the Toronto biotechnology company announced Monday, November 2. The funding is intended to advance its experimental Dualase gene-editing candidate toward initial clinical biomarker proof-of-concept in people with C9ORF72-associated ALS.
The company announcement said proceeds would support the first cohort of an adaptive Phase I/II trial. This is a financing milestone, not a report of successful treatment in patients. The release did not present human trial results, and the Dualase candidate is not an approved therapy.
Existing investors Lumira Ventures and adMare BioInnovations joined the round alongside Genesys Capital, the Business Development Bank of Canada’s Life Sciences Venture Fund, DeepWork Capital and Seido Capital. The company called the financing oversubscribed but did not disclose a valuation or investor ownership stakes.
Dualase is designed to shorten disease-causing repeat expansions in DNA while preserving normal sequences, according to Specific Biologics. Its initial target is a repeat expansion in the C9ORF72 gene, which the company identifies as a major inherited genetic cause of ALS and as associated with frontotemporal dementia.
Specific said the compact editing system can fit in a single adeno-associated virus delivery vehicle intended to reach affected motor neurons in the central nervous system. Its reported editing results come from patient-derived cell models and animal studies. Those preclinical findings do not establish safety, efficacy or a cure in people.
Chief Executive Brent Stead said the company aims to address disease at its genetic source. Hatteras partner Ben Scruggs cited the platform’s preclinical evidence and potential. Those assessments are company and investor views, rather than independent clinical confirmation.
The company sees potential applications across repeat-expansion disorders and said it has demonstrated preclinical editing in multiple neuromuscular targets. The funding announcement does not establish that the platform will work across those diseases or eliminate the risks associated with gene editing and delivery.
The next milestone described by the company is an early biomarker readout, not proof of a lasting functional benefit. The announcement did not provide a date for that readout. Clinical development will still need to determine whether the investigational treatment is safe and provides meaningful benefit to patients.
JBizNews Desk | Toronto
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