DYNE-251
- Generic Name
- DYNE-251
- Drug Type
- Biotech
- Background
DYNE-251 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1). It is being investigated for the treatment of Duchenne muscular dystrophy (DMD) in patients who are amenable to exon 51 skipping.
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Phase 1
Recruiting
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- Drug: Placebo
- First Posted Date
- 2022-09-01
- Last Posted Date
- 2025-01-22
- Lead Sponsor
- Dyne Therapeutics
- Target Recruit Count
- 88
- Registration Number
- NCT05524883
- Locations
- 🇧🇪
UZ Gent, Gent, Belgium
🇧🇪UZ Leuven, Leuven, Belgium
🇰🇷Pusan National University Yangsan, Yangsan-si, Gyeongsangnamdo, Korea, Republic of
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