BIIB147

BIIB147 is an investigational antisense oligonucleotide (ASO) being developed by Biogen, under a worldwide exclusive license from Ionis Pharmaceuticals, for the treatment of amyotrophic lateral sclerosis (ALS). The therapeutic targets stathmin-2 (STMN2) pre-mRNA to correct the cryptic splicing and premature polyadenylation that occurs due to TDP-43 pathology. In ALS, the nuclear clearance of TDP-43 leads to the inclusion of a cryptic exon in STMN2 pre-mRNA, resulting in a truncated, nonfunctional transcript and a subsequent loss of stathmin-2 protein, which is essential for axonal maintenance and repair. By binding to the STMN2 pre-mRNA, BIIB147 aims to restore normal splicing and rescue functional stathmin-2 protein levels. It is administered intrathecally and is currently in Phase 1/2 clinical development.