Wave Life Sciences is starting the FOCUS C9 clinical trial to test WVE-004, potentially a new medicine known as a “stereopure antisense oligonucleotide (ASO)”, for patients with a certain type of frontotemporal dementia (FTD) as well as amyotrophic lateral sclerosis (ALS). Research data suggest that WVE-004 has the potential to slow down disease progression because it may change the levels of toxic products of mutations in the C9ORF72 gene that is a common problem in both diseases.  In human cells with the C9ORF72 mutation and animals that produce the toxic products of the mutation, WVE-004 lowered these toxic products, while leaving the healthy protein produced by normal C9ORF72 unaffected, a treatment effect in animals that lasted for at least 6 months.  These results suggest that WVE-004 has the potential as a treatment for patients with FTD and ALS associated with C9ORF72 mutations and must be tested in a clinical trial.  The FOCUS C9 clinical trial will test the safety of WVE-004 and measure the impact on one of these toxic products (called “poly-GP”) in the spinal fluid, along with other symptoms which might predict future benefit for patients.