Specific is tackling serious genetic diseases such as C9ORF72-associated ALS by using its two-site Dualase editor to remove pathogenic repeat expansions at the DNA level, addressing a disease for which the company says there are no approved therapies. Its preclinical studies reported nearly complete collapse of the pathogenic repeat in patient-derived ALS cell models and correction of disease hallmarks in animal models with no detectable off-target effects, while the November 2026 US$35 million Series A is intended to move the lead candidate toward initial clinical biomarker proof-of-concept.