Wave next up in the C9orf72 pipeline

A week after Biogen and Ionis pulled the plug on their C9orf72-targeted antisense oligonucleotide, Wave announced that its phosphoryl guanidine-based oligo showed evidence of target engagement in a Phase I/II trial to treat C9orf72-associated amyotrophic lateral sclerosis and frontotemporal dementia. Four other companies are using diverse methods to reduce toxic C9orf72 mRNA.

The hexanucleotide repeat expansion (GGGGCC) in the C9orf72 gene is the most common genetic cause of ALS and FTD. It leads to build up of toxic transcripts and translation of aggregating dipeptide repeat proteins; both of which are thought to contribute to disease...