AOC 1042

AOC 1042 is an investigational antibody oligonucleotide conjugate (AOC) designed for the treatment of myotonic dystrophy type 1 (DM1). Developed by Avidity Biosciences (and associated with Novartis in the provided context), the drug utilizes a monoclonal antibody targeting the transferrin receptor 1 (TfR1) to deliver a small interfering RNA (siRNA) payload specifically to skeletal, cardiac, and smooth muscle cells. Once internalized via receptor-mediated endocytosis, the siRNA payload is intended to target and degrade the messenger RNA (mRNA) of the dystrophia myotonica protein kinase (DMPK) gene. By reducing the levels of toxic DMPK mRNA, AOC 1042 aims to address the underlying genetic cause of DM1, which is characterized by CTG repeat expansions that lead to RNA toxicity and progressive neuromuscular dysfunction. In the provided context, AOC 1042 is described as a late-stage candidate within a broader acquisition deal.