Toronto biotech developing Dualase genome editors as in vivo therapies, starting with C9ORF72 ALS
Specific Biologics is a Toronto-based biotechnology company developing in vivo genome-editing medicines built on its Dualase editors. Dualase uses a two-site mechanism, pairing an engineered Cas domain with an I-TevI nuclease domain, to enable precise removal, repair and insertion of DNA sequences, with an initial focus on removing expanded DNA repeats. Its pipeline spans CNS, lung and liver indications, delivered by single AAV or encapsulated all-RNA formats. In November 2026 it announced an oversubscribed $35 million Series A led by Hatteras Venture Partners to take its Dualase candidate into an adaptive Phase I/II trial for C9ORF72-associated ALS.