George Tachas, PhD, on Targeting Inflammation in DMD With Antisense Oligonucleotides


The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.

“We're seeking to stop the secondary causes of damage to muscles in DMD, which is the inflammation. The primary cause is dystrophin that leads to the loss of a structural protein, but then when you use the muscles, they contract, they get damaged, and then the immune system goes, hang on, this shouldn't be here andit's further damage to them. So we're targeting the secondary cause of immune mediated damage in Duchenne.”

ATL1102 regulated levels of genetic modifiers of disease progression and fibrosis in non-ambulant boys with Duchenne muscular dystrophy (DMD). These modifiers include sVCAM-1, LTBP4, BMP5, BMP6, IGF-I, and Thrombospondin-1, which ATL1102 modulated to closer to control levels (P <.0005).

These data, from a proteomic analysis of plasma from participants in a phase 2 trial (NCT05938023) of ATl1102, were presented by George Tachas, PhD, lead scientist, Percheron Therapeutics, at the 2024 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 3-6, in Orlando, Florida.

CGTLive® spoke with Tachas to learn more about ATL1102, which is an antisense oligonucleotide, and its mechanism of targeting inflammation as a secondary cause of muscle damage in patients with DMD. He outlined the current treatment landscape for DMD and posited that ATL1102 or a similar therapy may help fill gaps in treatment by targeting this cause of muscle damage.

Click here to view more coverage of the 2024 MDA Conference.

Tachas G, DeLisle R, Mueller C, et al. ATL1102 treatment of non-ambulant boys with DMD stabilizes function modifying plasma proteins with roles in immune, fibrosis, bone & growth physiology. Presented at: 2024 MDA Clinical and Scientific Conference; March 3-6; Orlando, FL. Poster #V401
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