Drugs / Zeleciment Rostudirsen
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05524883 | Aug 2022 → Nov 2029 expected | Duchenne muscular dystrophy | Dyne Therapeutics | Active not recruiting | No outcome recorded |
| Phase 31 trial | ||||||
| Phase 3 | NCT07608432 | Jun 2026 → Dec 2030 expected | Duchenne muscular dystrophy | Dyne Therapeutics | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 21
| Date | Issuer | Release |
|---|---|---|
| 2026-07-29 | Dyne Therapeutics | Regulatory Dyne Therapeutics Reports Second Quarter 2026 Financial Results and Recent Business Highlights dyne-tx.com ↗
Dyne’s BLA was accepted for review by the FDA in July 2026, with Priority Review granted (Prescription Drug User Fee Act (PDUFA) target action date set for January 21, 2027). |
| 2026-07-20 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD) dyne-tx.com ↗ |
| 2026-05-26 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD) dyne-tx.com ↗ |
| 2026-03-08 | Dyne Therapeutics | Results Dyne Therapeutics Announces New Positive Cardiopulmonary Results from DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD) dyne-tx.com ↗
Cardiopulmonary issues are a key area of concern in DMD, so we are particularly encouraged by new analyses showing improvement in both heart and lung function out to 24 months. |
| 2026-02-22 | Dyne Therapeutics | Results Dyne Therapeutics Announces Upcoming Presentations Across Neuromuscular Pipeline at 2026 MDA Clinical & Scientific Conference dyne-tx.com ↗ |
| 2025-12-08 | Dyne Therapeutics | Results Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD) dyne-tx.com ↗
The REC met its primary endpoint, demonstrating a statistically significant increase in muscle content-adjusted dystrophin expression to 5.46% of normal relative to baseline at six months (p<0.0001). |
| 2025-09-29 | Dyne Therapeutics | Regulatory Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy dyne-tx.com ↗
Dyne Therapeutics, Inc . (Nasdaq: DYN), a clinical-stage company, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. |
| 2025-08-04 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD) dyne-tx.com ↗
Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping. |
| 2025-04-24 | Dyne Therapeutics | Regulatory Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy dyne-tx.com ↗ |
| 2025-03-16 | Dyne Therapeutics | Results Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months dyne-tx.com ↗ |
| 2024-10-09 | Dyne Therapeutics | Results Dyne Therapeutics Presents Data at World Muscle Society Congress Highlighting Promise of FORCE™ Platform to Address Underlying Causes of Neuromuscular Diseases dyne-tx.com ↗
DYNE-251 demonstrated dose dependent exon skipping and dystrophin expression and improvement in multiple functional endpoints in both cohorts. |
| 2024-09-03 | Dyne Therapeutics | Results Dyne Therapeutics Announces New Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented Dystrophin Expression and Functional Improvement in Multiple Cohorts dyne-tx.com ↗ |
| 2024-05-20 | Dyne Therapeutics | Results Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints dyne-tx.com ↗
In Phase 1/2 DELIVER Trial, DYNE-251 Showed 3.2% Mean Unadjusted (7.6% Mean Muscle Adjusted) Dystrophin Expression at 6 Months in 10 mg/kg Cohort Administered Monthly |
| 2024-01-03 | Dyne Therapeutics | Results Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases dyne-tx.com ↗
In Phase 1/2 DELIVER Trial, DYNE-251 Showed 0.88% Mean Dystrophin Expression at 6 Months in 5 mg/kg Cohort Administered Monthly |
| 2023-03-23 | Dyne Therapeutics | Regulatory Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy dyne-tx.com ↗
DYNE-251, an investigational therapeutic for Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping, was granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations. |
| 2022-10-31 | Dyne Therapeutics | Regulatory Dyne Therapeutics Receives FDA Fast Track Designation for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy dyne-tx.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping. |
| 2022-10-11 | Dyne Therapeutics | Results Dyne Therapeutics Presents Preclinical Data from its Duchenne Muscular Dystrophy Programs Targeting Exons 51 and 53 at World Muscle Society 2022 Congress dyne-tx.com ↗ |
| 2022-07-05 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces FDA Clearance of IND Application for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy dyne-tx.com ↗ |
| 2022-01-18 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne Muscular Dystrophy dyne-tx.com ↗
Dyne Therapeutics, Inc . (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug Administration (FDA) has placed on clinical hold its Investigational New Drug (IND) application to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. |
| 2021-12-02 | Dyne Therapeutics | Regulatory Dyne Therapeutics Announces Submission of IND Application to Initiate Clinical Trial of DYNE-251 for Duchenne Muscular Dystrophy dyne-tx.com ↗
Dyne Therapeutics, Inc. (Nasdaq: DYN), a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to initiate a clinical trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to skipping exon 51. |
| 2021-10-01 | Dyne Therapeutics | Results Dyne Therapeutics Presents New In Vivo Data for its Duchenne Muscular Dystrophy Program During Muscle Study Group Annual Scientific Meeting Demonstrating Robust and Durable Exon Skipping and Dystrophin Expression dyne-tx.com ↗
DYNE-251 Achieves Exon 51 Skipping of 52% in the Diaphragm and 43% in the Heart in Non-Human Primates and is Well Tolerated in GLP Toxicology Study |
All press releases naming this drug 23 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Zeleciment Rostudirsen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07608432 ↗ |
| Known as | DYNE-251 | “Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks” NCT07608432 ↗ |
| Route | Intravenous | “Administered by IV infusion” NCT05524883 ↗ |