zeleciment

Zeleciment (DYNE-251 Antibody) is a humanized monoclonal antibody targeting TFRC (CD71). This product binds specifically to transferrin receptor 1 (TfR1) and facilitates the targeted delivery of conjugated nucleotide payloads (such as ASOs or PMOs) to muscle tissues via receptor-mediated endocytosis. It is a vital tool for researching the treatment of muscle diseases, including Duchenne muscular dystrophy (DMD) and myotonic dystrophy (DM1).

Zeleciment basivarsen (DYNE-101) is an antibody oligonucleotide conjugate (AOC), consisting of a Fab antibody named Zeleciment that binds to the transferrin receptor 1 (TfR1) on muscle cells, and an antisense oligonucleotide known as Basivarsen. This compound targets the RNA of the mutant dystrophia myotonica-protein kinase (DMPK) for RNase H-mediated degradation, and is utilized in the study of myotonic dystrophy type 1 (DM1).

Basivarsen linker is a connector employed in Zeleciment basivarsen, facilitating the attachment of a Fab antibody (Zeleciment) that targets the TfR1 receptor to an antisense oligonucleotide. Zeleciment basivarsen is studied for its potential application in the treatment of myotonic dystrophy type 1 (DM1).

Rostudirsen is an oligonucleotide and a component of Zelecimentrostudirsen. This compound is created by linking Rostudirsen with a fragment antibody known as Zeleciment. Zeleciment specifically binds to transferrin receptor 1 (TfR1), which is highly expressed in muscle tissue. Zelecimentrostudirsen targets muscle tissue, facilitating exon 51 skipping within cells, thus enabling muscle cells to produce dystrophin protein close to its full-length form.