Amyotrophic Lateral Sclerosis (ALS) is a fatal disease that produces progressive neurodegeneration. Great efforts have been dedicated to find a cure for this disease with no success up to date.

The precise molecular mechanisms responsible of ALS remain unknown. The most frequent genetic cause of ALS is a mutation in the C9ORF72 gene. This gene contains a sequence of six nucleotides (5’-GGGGCC-3’/3’-CCCCGG-5’) which appears massively repeated in a high proportion of ALS patients.

It has been proposed that this defect in C9ORF72 gene contributes to the disease through the toxicity mediated by transcription of RNA containing the expansion of the repeated motif.

Combat_ALS action aims to develop molecules to counteract the deleterious effects produced by the presence of RNAs containing the repeat expansion of C9ORF72. To that end, two kinds of molecules are being developed: (I) antisense oligonucleotides (ASO) and (II) small organic compounds with affinity for the RNAs.