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

Commentary
Video

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.

REFERENCE
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
Related Videos
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level
Caspian Oliai, MD, MS, the medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center
Frederick “Eric” Arnold, PhD
Genovefa (Zenia) Papanicolaou, MD, an infectious diseases specialist at Memorial Sloan Kettering Cancer Center
Jeffrey Chamberlain, PhD, on Exciting New Research at MDA 2024
Alan Beggs, PhD, on Challenges in Therapeutic Development for Rare Diseases
Akshay Sharma, MBBS, a bone marrow transplant physician at St. Jude Children’s Research Hospital
PJ Brooks, PhD
John DiPersio, MD, PhD, the director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine
© 2024 MJH Life Sciences

All rights reserved.