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Zeleciment rostudirsen (z-rostudirsen)

Duchenne muscular dystrophy

Also known as DYNE-251
Manufacturer
Dyne Therapeutics
Regulatory submission
BLA submitted May 2026
Last updated
78 sources

Product information and disease descriptionProduct descriptionProduct informationPharmacology

Pharmacodynamics

6 evidence topics · 6 sources

Preclinical FORCE–M23D evidence

“Importantly, FORCE–M23D treatment resulted in improved functional outcomes compared with administration of unconjugated M23D.” (opens the source at this quote in a new tab)

FORCE platform toxicology: repeat-dose studies in cynomolgus monkeys

“Reticulocytes were transiently decreased 24-72 hrs after administration of either FORCE-PMO or FORCE-ASO but not FORCE-siRNA” (opens the source at this quote in a new tab)

FORCE-M23D and CNS outcomes: hTfR1/mdx mice

“A single dose of FORCE-M23D leads to durable resolution of anxiety in hTfR1/mdx mice” (opens the source at this quote in a new tab)

FORCE-M23D muscle pathology: D2-mdx mouse model

“FORCE-M23D achieved robust dystrophin restoration to the sarcolemma of skeletal muscles and improved muscle morphology of D2-mdx mice.” (opens the source at this quote in a new tab)

DYNE-251 preclinical exon skipping: nonhuman primates

“DYNE-251 led to pronounced exon 51 skipping in the heart, diaphragm and quadriceps 8 weeks after the first dose” (opens the source at this quote in a new tab)

DELIVER: dose-dependent target engagement

“DYNE-251 Demonstrated Dose-Dependent Exon Skipping and Delivery of PMO to Muscle” (opens the source at this quote in a new tab)