Current therapeutic attempts to treat DMD include viral-mediated microdystrophin gene replacement, exon skipping and nonsense suppression therapy. However, toxicity problems
and off-targets effects have arisen from those therapies. Symptomatic therapies to target muscle ischemia, enhance muscle regeneration, and reduce inflammation are also under investigation. However, these are not treating the cause of the disease.
At Consortium.AI, we are developing bioavailable small chemical compounds with exon-skipping function. We aim to target both the underlying genetic mutations as well as the secondary complications, alone or in combination with other treatments, with the goal to slow
the disease progression and provide more definitive treatment options for DMD.