Webinars
Date: January 15, 2026
Time: 11am-12pm EST / 5-6pm CET
Title: Sustained muscle function improvement after a single in vivo dose of antibody oligonucleotide conjugate (AOC) in the mdx mouse model for Duchenne muscular dystrophy
Description:
Duchenne muscular dystrophy results from the absence of dystrophin and exon skipping aims to restore a shorter, partially functional form of the protein. Antisense oligonucleotide–mediated exon skipping has FDA approval, but current dystrophin restoration remains low, warranting improved approaches. This study evaluated antibody-oligonucleotide conjugates (AOCs) designed for targeted muscle uptake in mdx mice using exon 23-specific AONs. Mice received a single intravenous injection containing either vehicle (control) or different AONs (eg. DAR4-conjugates) at different doses and underwent repeated functional testing over 16 weeks. Results showed sustained improvement in multiple muscle function tests, increased levels of exon skipping and dystrophin protein restoration in the mdx mice when AONs are conjugated to an antibody using GlycoConnect® technology.
Speaker:
Title: RNA-PROTACs for Targeted Degradation of TDP-43
Description:
Proteolysis-targeting chimeras (PROTACs) exploit the endogenous ubiquitin–proteasome system to induce protein degradation. RNA-PROTACs are oligonucleotide conjugates that bring RNA-binding proteins and E3 ligases into proximity, leading to proteasomal degradation of the RNA-binding protein. We rationally designed an RNA-PROTAC that binds the ALS-associated RNA-binding protein TDP-43, which reduces TDP-43 protein levels in cells.
