$35 million to edit ALS out at its root: backers bet on a therapy that targets the cause, not the symptoms

ALS slowly takes away the things most of us never think about: walking, speaking, swallowing, breathing. For many families it runs in the genes, and the most common inherited cause is a kind of stutter in the DNA — a short stretch of the C9ORF72 gene repeated far too many times. Toronto-based Specific Biologics has built a gene-editing tool, Dualase, designed to shorten that repeated stretch while leaving the healthy DNA around it untouched. In lab tests using cells grown from patients, it shrank the faulty repeat almost completely, with no detectable edits in the wrong places. Now investors have put US$35 million behind it — more than the company asked for — to carry the treatment into its first clinical proof-of-concept tests in people with this form of ALS, for which no approved therapy yet targets the genetic cause.

SDG 3 · Good health and well-being — Mechanism: funding & finance