Next week at the American Academy of Neurology Meeting (AAN) in Philadelphia, Biogen will present promising results of the phase 1/2 study of its newly named investigational therapy tofersen (previously BIIB067), which is now enrolling in a phase 3 trial. Tofersen is an antisense oligonucleotide (ASO), a type of designer DNA drug, targeting SOD1.
The ALS Association is proud to be the first funder of antisense technology. Our support dates back to 2004, when antisense was just an idea in Dr. Don Cleveland’s lab at University of California San Diego (UCSD). This new potential treatment targets the second most common cause of inherited ALS, mutations in the SOD1 gene.