zeleciment rostudirsen
z-rostudirsen · DYNE-251 Nucleic Acid Target: TfR1, dystrophin exon 51, dystrophin, exon 51
Most recent annual filing: 2026-03-02
TfR1-targeting Fab-conjugate delivery platform that links therapeutic payloads (PMOs, ASOs, siRNAs, enzymes) to a proprietary anti-TfR1 Fab via a clinically-validated linker, enabling targeted delivery to skeletal, cardiac and smooth muscle and CNS. Disease focus: neuromuscular diseases including DMD, DM1, FSHD, Pompe disease, rare skeletal muscle diseases, and cardiac/metabolic muscle diseases.
Targets: TfR1, transferrin receptor 1
Articles open on Substack. Some deep dives are subscriber-only.