atesidorsen

Atesidorsen (ATL1103) is a second-generation **antisense oligonucleotide** designed to target and inhibit the translation of human **growth hormone receptor (GHR) mRNA**, leading to reduced levels of growth hormone (GH) receptor protein and lower concentrations of **insulin-like growth factor-I (IGF-I)** in the bloodstream. The drug comprises 20 nucleotides with a phosphorothioate backbone and 2′-O-methoxyethyl modifications at both ends, improving plasma half-life and mRNA affinity. The therapeutic rationale is based on the inhibition of IGF-I production via downregulation of the GHR pathway, primarily developed for **acromegaly** and other diseases associated with excessive GH and IGF-I, such as diabetic retinopathy, diabetic nephropathy, and certain cancers. The drug is administered subcutaneously and has shown proof of concept in phase 2 trials for acromegaly, significantly lowering IGF-I levels in patients with a favorable safety profile, most commonly mild to moderate injection site reactions[1][3][5][6][7][12][14].

Other names

ATL1103ATL-1103ATL 1103atesidorsen