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Also known as DYNE-251, Zeleciment Rostudirsen (DYNE-251)
Zeleciment rostudirsen · 2 trials · 13 indications
| Arm | Type | Description |
|---|---|---|
| Placebo-Controlled Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | Participants will be randomized to receive zeleciment rostudirsen, once every 4 weeks (Q4W) for up to 72 weeks. |
| Placebo-Controlled Period: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo, Q4W for up to 72 weeks. |
| Open-Label Long-Term Extension Period: Zeleciment Rostudirsen (DYNE-251) | EXPERIMENTAL | All participants who complete the Placebo-Controlled Period of the study will receive zeleciment rostudirsen administered Q4W for up to 96 weeks. |
| Placebo-Controlled MAD Period - DYNE-251 | EXPERIMENTAL | DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks. |
| Placebo-Controlled MAD Period - Placebo | EXPERIMENTAL | Placebo will be administered Q4W or Q8W over 24 weeks. |
| Open-Label and Long-Term Extension Period - DYNE-251 | EXPERIMENTAL | DYNE-251 will be administered Q4W or Q8W for up to 288 weeks after participants complete the Placebo-Controlled MAD Period of the study. |
| Name | Type | Description |
|---|---|---|
| Zeleciment Rostudirsen (DYNE-251) | DRUG | Administered by IV infusion |
| Placebo | DRUG | Administered by IV infusion |
| DYNE-251 | DRUG | Administered by IV infusion |
Inclusion Criteria: * Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping . * Rise From Floor (RFF) time must be \< 10 seconds for both screening assessments . * Receiving a stable daily or weekend do...
Zeleciment rostudirsen is an investigational RNA therapy being developed for Duchenne muscular dystrophy (DMD). It is designed for patients whose disease is amenable to exon 51 skipping. The program is in late-stage development and has not been approved by the FDA.
Zeleciment rostudirsen targets transferrin receptor 1 (TfR1). The therapy uses a TfR1-binding antibody fragment to deliver an exon-skipping payload to muscle tissue. This targeted delivery approach is intended to increase exposure of the RNA payload in skeletal and cardiac muscle.
Zeleciment rostudirsen is being developed by Dyne Therapeutics, Inc., which trades on the Nasdaq under the ticker DYN. Dyne Therapeutics is the sponsor of the clinical program evaluating the drug in Duchenne muscular dystrophy.
Zeleciment rostudirsen is in the NDA/BLA stage of development. Two clinical trials are active: a Phase 3 trial and a Phase 1 trial. The drug is investigational and has not been approved by the FDA for any indication.
Zeleciment rostudirsen is being evaluated in two clinical trials. The Phase 3 FORZETTO trial (NCT07608432) is recruiting ambulatory participants with Duchenne muscular dystrophy in the United States. The Phase 1 trial (NCT05524883) is active but not recruiting and has enrolled participants across the United States, Australia, Belgium, Canada, Ireland, Italy, South Korea, Spain, and the United Kingdom.
Yes, Zeleciment rostudirsen is also known as DYNE-251. DYNE-251 is the investigational code name used by Dyne Therapeutics for the same RNA therapy being developed for Duchenne muscular dystrophy. Both names refer to the identical drug candidate.