zeleciment basivarsen

Zeleciment basivarsen is an investigational antibody-oligonucleotide conjugate (AOC) designed for the treatment of myotonic dystrophy type 1 (DM1). It consists of an antisense oligonucleotide (basivarsen) conjugated to an antigen-binding fragment (zeleciment) that binds to the transferrin receptor 1 (TfR1)[1][2]. This design enables delivery to both muscle and the central nervous system. The drug works by reducing toxic nuclear DMPK RNA to release splicing proteins and allow normal mRNA processing, thereby addressing the central pathobiology of DM1[1][3]. Zeleciment basivarsen has been granted Breakthrough Therapy, Orphan Drug, and Fast Track designations by the U.S. FDA, and Orphan Drug designation by the European Medicines Agency[1]. The drug is being developed by Dyne Therapeutics and is currently in the Phase 1/2 ACHIEVE clinical trial, with a selected registrational dose of 6.8 mg/kg administered every 8 weeks (Q8W)[1][2][8].

Other names

z-basivarsen