Our lead product candidate, DYN101, is an antisense oligonucleotide or ASO, designed to reduce the expression of dynamin 2 or DNM2, for the treatment of CNM. DNM2 is a protein, which when elevated, is toxic in muscle cells.
Our co-founder and Chief Scientific Officer, Dr. Belinda Cowling, conducted the foundational research that identified the central role that the DNM2 protein plays in disease pathogenesis in several forms of CNM, and that the down regulation of the expression of DNM2 could provide a novel therapeutic approach. DNM2 is an enzyme present in all cells of the body that is involved in membrane remodeling. We have demonstrated that increased DNM2 is toxic to muscle cells and is the principal cause of disease in all three classical forms of CNM, which make up approximately 72% of the CNM patient population in the United States, European Union, Japan and Australia.
There are three classical forms of CNM. The most severe form of CNM is X-linked myotubular myopathy, or XLCNM, caused by mutations in the MTM1 gene, which accounts for approximately 57% of CNM patients. Mutations in the DNM2 gene result in autosomal dominant CNM, or ADCNM (approximately 11% of CNM patients), and mutations in the BIN1 gene result in autosomal recessive CNM, or ARCNM (approximately 4% of CNM patients).