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Watch George Tachas, PhD, lead scientist Percheron Therapeutics Limited discuss research on antisense oligonucleotide therapies in mouse models of #DMD

ATL1102 (Percheron Therapeutics) antisense oligonucleotide (ASO) therapy has shown potential benefit in treating Duchenne muscular dystrophy (DMD), both in human and mice studies. The company presented multiple posters at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida, on ATL1102. One such poster looked at the combination of ASO therapy with a dystrophin morpholino exon 23 skipping restoration drug (PMO) in mice models and found that the ASO showed benefits in muscle function both in combination with the PMO and alone.

CGTLive® spoke with George Tachas, PhD, senior author of the poster and lead scientist, Percheron Therapeutics, to learn more about ATL1102, the murine studies presented on, and how these findings may translate into the ASO’s benefit in human patients with DMD. He emphasised that the ASO therapy is tackling inflammation as a secondary cause of damage in DMD and therefore may synergise well with therapies targeting the primary, genetic cause of damage.

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