regulatory
confidence high
sentiment positive
materiality 0.70
FDA lifts clinical hold on Dyne's IND for DYNE-251 in Duchenne muscular dystrophy
Dyne Therapeutics, Inc.
- FDA cleared IND and lifted clinical hold for DYNE-251, targeting exon 51 skipping in DMD.
- Dyne expects to begin dosing patients in a Phase 1/2 trial in mid-2022.
- Trial will enroll 30–50 males ages 4–16, both ambulant and non-ambulant.
- Endpoints include safety, dystrophin expression by Western blot, PK/PD, and muscle function.
- Company will release trial design details and data timing upon first dosing.