George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles

“[We’re exploring this] as a monotherapy... because some boys don't have the exon splicing drugs available for that type of genetic condition...We're hoping [these findings] translate also into maintaining ambulation. And there's some interesting talks on some of this work [in DMD] and we look forward to hearing what other people are doing in the gene therapy space and looking to see how we can work with other people to, while they tackle the primary cause, we tackle the secondary cause and do it better than the corticosteroids.”

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 emphasized that the ASO therapy is tackling inflammation as a secondary cause of damage in DMD and therefore may synergize well with therapies targeting the primary, genetic cause of damage.

REFERENCE

Padhye A, Houweling PJ, Wilson T, et al. Mdx mice dosed with antisense to CD49d & dystrophin exon skip morpholino; improved muscle force & affected pathways support ATL1102 combination in DMD. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6: Orlando, Florida. Popster #V410