This section highlights FDA-related milestones and regulatory updates for drugs developed by Dyne Therapeutics (DYN).
Over the past two years, Dyne Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
DYNE-101, DYNE-251, and DYNE-302. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
DYNE-101 - FDA Regulatory Timeline and Events
DYNE-101 is a drug developed by Dyne Therapeutics for the following indication: Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DYNE-101
- Announced Date:
- June 17, 2025
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1).
AI Summary
Dyne Therapeutics, Inc. has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for its investigational drug DYNE-101, aimed at treating myotonic dystrophy type 1 (DM1). This designation is reserved for drugs that show early clinical promise in treating serious conditions, allowing for enhanced guidance and potentially faster review processes by the FDA.
Along with the designation, Dyne has submitted a revised protocol for the Registrational Expansion Cohort of its ACHIEVE trial. The updated protocol now elevates video hand opening time (vHOT) as the primary endpoint based on new long-term data, which suggests vHOT is an effective early indicator of clinical benefit in DM1 patients. The cohort will enroll 60 participants at multiple U.S. sites, and the data gathered will aid in pursuing U.S. Accelerated Approval for DYNE-101.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- February 27, 2025
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc. reported recent business highlights.
AI Summary
Dyne Therapeutics shared key business updates, highlighting promising progress on their neuromuscular disease therapies. The company reported strong clinical data from the ACHIEVE trial of DYNE-101 in myotonic dystrophy type 1 (DM1), which showed significant functional benefits and safety. Based on these positive results, Dyne is rapidly moving to fully enroll the Registrational Expansion Cohort, planned for mid-2025, to support a submission for U.S. Accelerated Approval. Additionally, the company is advancing its DELIVER trial for DYNE-251 in Duchenne muscular dystrophy (DMD). Full enrollment of its Registrational Expansion Cohort, comprising about 32 patients, is expected in the first quarter of 2025, with data due later that year to support another potential submission. Dyne’s CEO, John Cox, emphasized the importance of these results in addressing the underlying biology of these devastating diseases and paving the way for transformational therapies.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- January 21, 2025
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1 (DM1).
AI Summary
Dyne Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its investigational drug DYNE-101, aimed at treating myotonic dystrophy type 1 (DM1). This rare genetic disorder causes muscle weakness, breathing problems, and other serious symptoms, and currently, there are no approved treatments for it. The Fast Track status is given to drugs that address serious medical conditions and meet an unmet need, which can lead to more frequent interactions with the FDA and an expedited review process through a rolling submission of data. The designation comes after promising results from the ongoing Phase 1/2 ACHIEVE clinical trial, where DYNE-101 showed significant clinical benefits and effects on a key disease biomarker. The company hopes this will fast-track DYNE-101’s development to provide patients with a potentially transformative therapy as soon as possible.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- January 10, 2025
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc. announced new clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1).
AI Summary
Dyne Therapeutics recently shared new clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1). The data showed that DYNE-101 has a significant impact on key disease biomarkers, including a noticeable reduction in DMPK RNA levels and marked splicing correction. Patients experienced early and sustained functional improvements in measures such as muscle strength, myotonia, and mobility. Additionally, positive trends were observed in patient-reported outcomes, including improvements in central nervous system-related symptoms. Based on these encouraging results, the company has selected a 6.8 mg/kg dose, administered every 8 weeks, for the upcoming global Registrational Expansion Cohort. This cohort aims to gather further functional and biomarker data, potentially supporting a submission for U.S. Accelerated Approval in H1 2026, and represents a critical step toward a transformative treatment for DM1 patients.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- May 20, 2024
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc. announced positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1)
AI Summary
Dyne Therapeutics has announced positive results from its ongoing Phase 1/2 ACHIEVE trial evaluating DYNE-101 for patients with myotonic dystrophy type 1 (DM1). In the trial’s 5.4 mg/kg cohort, patients achieved a 27% mean splicing correction at 3 months, demonstrating a strong, dose-dependent effect on the genetic markers of the disease. These findings were accompanied by improvements in key functional measures, including reductions in myotonia, enhanced muscle strength, and better performance in timed function tests. Moreover, patient-reported outcomes, as measured by DM1-ACTIVc and MDHI, revealed noticeable enhancements in daily activities and overall quality of life. The data suggest that DYNE-101 could restore normal mRNA processing and potentially slow the progression of DM1, positioning it as a promising therapeutic option for those affected by this challenging condition.
Read Announcement
DYNE-251 - FDA Regulatory Timeline and Events
DYNE-251 is a drug developed by Dyne Therapeutics for the following indication: Duchenne Muscular Dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DYNE-251
- Announced Date:
- April 24, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD).
AI Summary
Dyne Therapeutics announced that the European Commission has granted orphan drug designation for its experimental therapy DYNE-251, which is aimed at treating Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. This designation highlights DYNE-251’s potential to address rare, life-threatening diseases affecting fewer than five in 10,000 people. Under this status, the company may benefit from reduced regulatory fees, clinical protocol assistance, and up to 10 years of market exclusivity if the therapy is approved. The decision comes as DYNE-251 is being evaluated in the Phase 1/2 DELIVER clinical trial, where early data have shown sustained functional improvements over 18 months. With the registrational expansion cohort fully enrolled, Dyne Therapeutics plans to present more detailed results in late 2025 and aims to move towards regulatory submissions by early 2026.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- April 24, 2025
- Estimated Event Date Range:
- October 1, 2025 - December 31, 2025
- Target Action Date:
- LATE 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc announced that Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025
AI Summary
Dyne Therapeutics recently announced important progress in their clinical trial of DYNE-251 for Duchenne muscular dystrophy. The DELIVER trial has shown sustained functional improvements over 18 months, and the fully enrolled registrational expansion cohort, which includes 32 patients, has now completed enrollment. Dyne plans to share the data from this cohort in late 2025. This anticipated data release is a critical milestone that could help support regulatory submissions for the treatment in early 2026. The upcoming results may strengthen the case for DYNE-251 as a promising therapeutic option for individuals with genetically driven neuromuscular disease, offering hope for significant improvements in patient function and quality of life.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- March 16, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that Potential for Biologics License Application submission for U.S. accelerated approval in early 2026 -
AI Summary
Dyne Therapeutics, Inc. announced promising updates from its ongoing Phase 1/2 DELIVER trial for DYNE-251, a therapy developed for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The study has shown sustainable improvements in muscle function and maintained a favorable safety profile. The registrational expansion cohort is fully enrolled, and the company expects to share these important findings later in 2025.
Based on these strong clinical results and unprecedented dystrophin expression data, Dyne is planning a submission of a Biologics License Application for U.S. accelerated approval in early 2026. This potential submission follows a well-established regulatory pathway that uses increased dystrophin levels as a surrogate endpoint, offering new hope for faster access to a therapy that meets the urgent need of DMD patients.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- March 16, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg/kg Q4W (approximate PMO dose).
AI Summary
Dyne Therapeutics announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial. The study showed that patients with Duchenne muscular dystrophy (DMD) who received DYNE-251 at the selected registrational dose of 20 mg/kg every four weeks experienced unprecedented, sustained functional improvement. This improvement was measured through multiple functional endpoints, highlighting the therapy’s potential to address unmet needs in patients amenable to exon 51 skipping.
The data also confirmed a favorable safety profile, with no new treatment-related serious adverse events reported. Additionally, the observed near-full length dystrophin expression supports the clinical efficacy of DYNE-251. These positive results are expected to accelerate the development pathway, with an anticipated Biologics License Application submission for U.S. accelerated approval in early 2026.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- October 9, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that previously reported clinical and preclinical data across its pipeline will be featured in poster presentations at the 29th Annual Congress of the World Muscle Society, held virtually and in Prague, Czech Republic, October 8-12, 2024.
AI Summary
Dyne Therapeutics, Inc. announced that previously reported clinical and preclinical data from its pipeline will be featured in poster presentations at the 29th Annual Congress of the World Muscle Society, held virtually and in Prague, Czech Republic, from October 8-12, 2024. This opportunity showcases the FORCE™ platform, which aims to deliver targeted therapies for neuromuscular diseases by reaching key tissues such as skeletal, cardiac, smooth muscle, and the central nervous system.
The posters will include clinical data from DYNE-251 and DYNE-101, as well as preclinical research in facioscapulohumeral muscular dystrophy and Pompe disease. The presentations highlight the potential of the FORCE™ platform to provide innovative, life-transforming treatments for patients with genetically driven muscle disorders, demonstrating promising advances in both clinical and preclinical studies.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- September 3, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced new clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping demonstrating unprecedented dystrophin expression and functional improvement in multiple cohorts.
AI Summary
Dyne Therapeutics released promising clinical data from its Phase 1/2 DELIVER trial with DYNE-251, a treatment designed for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The data revealed unprecedented dystrophin expression—levels reportedly more than 10 times higher than those seen with existing treatments—marking the highest level ever documented for an exon 51 skipping therapy.
In addition to the robust biomarker response, patients showed meaningful improvements in muscle function, including improvements in walking speed and other key physical performance measures. These results suggest that DYNE-251 effectively reaches vital muscle groups such as the heart, diaphragm, and skeletal muscles, translating preclinical benefits into real improvements for patients. With these encouraging findings, Dyne plans to initiate registrational cohorts in the DELIVER trial and update its path to registration by the end of 2024.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- September 3, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that Initiating Registrational Cohorts with Update on Path to Registration by Year-End 2024 -
AI Summary
Dyne Therapeutics recently announced that it will begin registrational cohorts in its ongoing Phase 1/2 DELIVER trial for DYNE-251. The company shared promising clinical data showing that the treatment achieved unprecedented levels of dystrophin expression and improved functional outcomes in patients with Duchenne muscular dystrophy amenable to exon 51 skipping. These results include significant improvements in digital mobility measures, which are important for patient daily activity and have met established international thresholds.
Dyne plans to use this data to initiate registrational cohorts and pursue expedited approval pathways. An update on the path to registration is expected by the end of 2024. This milestone demonstrates the company’s commitment to advancing its innovative treatments and transforming the treatment landscape for patients with genetically driven muscle diseases.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- May 20, 2024
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc announced positive clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics, Inc. announced positive clinical results from its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In the 10 mg/kg cohort, patients showed a mean unadjusted dystrophin expression of 3.2% of normal, which, when adjusted for muscle content, reached 7.6%—a level 10 times higher than reported for the standard of care given weekly. These encouraging findings were accompanied by trends of functional improvements, including benefits in the North Star Ambulatory Assessment (NSAA), faster time to rise from the floor, improved 10-meter walk/run times, and better stride velocity.
The therapy also demonstrated a favorable safety profile, and the enrollment for the 40 mg/kg cohort was completed. Overall, these data support DYNE-251’s potential as a transformative treatment option for DMD patients who are amenable to exon 51 skipping.
Read Announcement
DYNE-302 - FDA Regulatory Timeline and Events
DYNE-302 is a drug developed by Dyne Therapeutics for the following indication: In Facioscapulohumeral Muscular Dystrophy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DYNE-302
- Announced Date:
- June 5, 2025
- Indication:
- In Facioscapulohumeral Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that it will be presenting new preclinical data demonstrating the potential of DYNE-302 to achieve functional improvement in facioscapulohumeral muscular dystrophy (FSHD).
AI Summary
Dyne Therapeutics announced that it will present new preclinical data at the 32nd Annual FSHD Society’s International Research Congress. The findings show that DYNE-302, when given as a single intravenous dose at the peak of muscle weakness in a severe FSHD mouse model, restored the animals’ ability to run on a treadmill. This result suggests a functional improvement in muscle performance.
Further analysis revealed that the therapy corrected gene expression related to muscle damage and inflammation in skeletal muscle. These outcomes indicate that targeting DUX4 mRNA with DYNE-302 may reverse existing muscle disease in patients suffering from facioscapulohumeral muscular dystrophy (FSHD). The data, presented by Dr. Stefano Zanotti, head of neuromuscular research at Dyne, supports the potential of DYNE-302 as a promising treatment option for this rare, progressive disorder.
Read Announcement
