Dyne Therapeutics (DYN) has upcoming FDA regulatory milestones for DYNE-101. The table below outlines estimated target dates and event types for these pending regulatory actions.
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-253, DYNE-302, z-rostudirsen, and DYNE-251. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
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:
- September 8, 2026
- Target Action Date:
- Q1 2027
- Estimated Target Date Range:
- January 1, 2027 - March 31, 2027
- 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 Topline data from the ACHIEVE REC are on track for Q1 2027 -
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- September 8, 2026
- 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 it will present additional one-year clinical data from the Phase 1/2 ACHIEVE trial of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) for DM1 at the 31st Annual International Congress of the World Muscle Society (WMS) being held September 29, 2026, through October 3, 2026, virtually and in Hiroshima, Japan, as well as the 2026 Annual Meeting of the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) being held September 29, 2026, through October 2, 2026, virtually and in Orlando, Florida.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- June 3, 2026
- Indication:
- Designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus
Announcement
Dyne Therapeutics, Inc. announced the completion of enrollment in the registrational expansion cohort (REC) of the Phase 1/2 ACHIEVE trial of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in individuals with DM1.
AI Summary
Dyne Therapeutics said it has finished enrolling the registrational expansion cohort of its Phase 1/2 ACHIEVE trial for zeleciment basivarsen, also called z-basivarsen or DYNE-101, in people with myotonic dystrophy type 1 (DM1). The cohort includes 71 participants, marking an important step in the company’s development plan for the treatment.
The company said topline data from this cohort are expected in the first quarter of 2027. This update shows that the trial is moving forward as planned and may help determine the next stage of development for the drug candidate.
DM1 is a genetic disease that can affect muscles and other parts of the body, causing weakness and other long-term symptoms. Dyne Therapeutics is studying z-basivarsen as a potential therapy designed to address the needs of people living with this condition.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- January 20, 2026
- 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 Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for zeleciment basivarsen (z-basivarsen), for the treatment of myotonic muscular dystrophy type 1 (DM1).
AI Summary
Dyne Therapeutics announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation to zeleciment basivarsen (z‑basivarsen) for the treatment of myotonic dystrophy type 1 (DM1). In Japan, this designation applies to drugs for rare diseases affecting fewer than 50,000 patients and can bring development subsidies and up to 10 years of market exclusivity if approved.
Z‑basivarsen is being evaluated in the global Phase 1/2 ACHIEVE trial and has shown early, sustained improvements in myotonia, muscle strength, and functional measures, with a favorable safety profile. Dyne expects to complete enrollment in the ACHIEVE Registrational Expansion Cohort in early Q2 2026. The registrational cohort is designed to support potential regulatory submissions and measures myotonia change at six months.
The medicine is an antisense oligonucleotide linked to an antibody fragment that targets the transferrin receptor to deliver drug to muscle and the central nervous system. It aims to reduce toxic DMPK RNA and restore normal RNA processing in people with DM1.
Read Announcement- Drug:
- DYNE-101
- Announced Date:
- October 6, 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 additional one-year data from its ongoing Phase 1/2 ACHIEVE clinical trial of zeleciment basivarsen (z-basivarsen, formerly known as DYNE-101), in patients with myotonic dystrophy type 1 (DM1) demonstrating clinically meaningful improvements in function and strength at the selected registrational dose.
AI Summary
Dyne Therapeutics presented one-year data from the Phase 1/2 ACHIEVE trial of z-basivarsen (formerly DYNE-101) in patients with myotonic dystrophy type 1 at the selected registrational dose (6.8 mg/kg every eight weeks). Participants showed robust and sustained improvements in muscle stiffness (myotonia) as measured by video hand opening time and key functional tests, including the 10-Meter Walk/Run, 5-Times Sit-to-Stand, and 9-Hole Peg Test.
Muscle strength improved significantly across both upper and lower limbs, as measured by Quantitative Muscle Testing of hand grip, elbow flexion/extension, ankle dorsiflexion, and knee flexion/extension. Patient-reported outcomes highlighted meaningful benefits in cognitive function, sleep, fatigue, communication, emotional health, pain, mobility, and daily activities. Both Patient and Clinician Global Impression of Change scales indicated reduced overall disease burden. Z-basivarsen also maintained a favorable safety profile with no serious related adverse events, supporting its potential to deliver broad functional improvements in myotonic dystrophy type 1.
Read Announcement- 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
DYNE-253 FDA Regulatory Events
DYNE-253 is a drug developed by Dyne Therapeutics for the following indication: for 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-253
- Announced Date:
- September 3, 2026
- Indication:
- for Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that it will present preclinical data supporting the ongoing development of DYNE-253, DYNE-245, DYNE-244 and DYNE-255 for the potential treatment of DMD amenable to skipping of exons 53, 45, 44 and 55, respectively, at the 27th Annual Neuromuscular Study Group (NMSG) Scientific Meeting being held September 25-27, 2026, in San Antonio, TX.
Read Announcement
DYNE-302 FDA Regulatory 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:
- July 28, 2026
- Indication:
- In Facioscapulohumeral Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for its IND application to initiate a Phase 1 clinical trial for DYNE-302 in facioscapulohumeral muscular dystrophy (FSHD). .
AI Summary
Dyne Therapeutics, Inc. announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug application for DYNE-302, allowing the company to begin a Phase 1 clinical trial in people with facioscapulohumeral muscular dystrophy (FSHD). DYNE-302 is the company’s third clinical program using its FORCE platform, which is designed to deliver treatments to muscle tissue and address rare neuromuscular diseases.
The first study cohort will include nine participants. They will receive three intravenous doses every four weeks and will be randomly assigned in a 2-to-1 ratio to DYNE-302 at 1.5 mg/kg or placebo. After this cohort, Dyne plans to test higher doses and less frequent treatment schedules. Participants who complete the placebo-controlled portion may join a long-term extension study and receive DYNE-302 for up to 96 additional weeks. The company aims to pursue traditional U.S. approval for the treatment.
Read Announcement- 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
Z-rostudirsen FDA Regulatory Events
Z-rostudirsen is a drug developed by Dyne Therapeutics for the following indication: myotonic dystrophy type 1 (DM1) and Pompe disease.
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:
- z-rostudirsen
- Announced Date:
- July 20, 2026
- Indication:
- myotonic dystrophy type 1 (DM1) and Pompe disease
Announcement
Dyne Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics said the U.S. Food and Drug Administration has accepted its Biologics License Application for zeleciment rostudirsen, also called z-rostudirsen or DYNE-251, for people with Duchenne muscular dystrophy who are able to skip exon 51. The company is seeking accelerated approval, using dystrophin as the main measure to support the review.
Dyne said results from the DELIVER trial’s registrational expansion cohort showed that treatment every four weeks led to a strong and statistically significant rise in dystrophin production. The company also reported signs of functional improvement across several clinical measures, along with a favorable safety profile. The FDA has granted Priority Review, and the review process is now underway.
Dyne is also developing other DMD treatment candidates aimed at additional exon-skipping targets, as part of a broader pipeline for the disease.
Read Announcement- Drug:
- z-rostudirsen
- Announced Date:
- May 26, 2026
- Indication:
- myotonic dystrophy type 1 (DM1) and Pompe disease
Announcement
Dyne Therapeutics, Inc. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) 20 mg/kg Q4W for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics has submitted a Biologics License Application to the U.S. Food and Drug Administration for zeleciment rostudirsen, also called z-rostudirsen or DYNE-251, to treat people with Duchenne muscular dystrophy who are able to skip exon 51. The company is seeking Accelerated Approval, using dystrophin as a surrogate marker for benefit. In its DELIVER trial expansion cohort, the treatment showed a strong and statistically significant rise in dystrophin production, along with signs of functional improvement across several clinical measures. Dyne also reported a favorable safety profile. The proposed dose is 20 mg/kg given by intravenous infusion once every four weeks. Dyne has asked for Priority Review, which could shorten the FDA review time if granted. The company says it still expects a possible U.S. launch in the first quarter of 2027, depending on the FDA’s decision timeline.
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:
- May 20, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced the initiation of the Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics announced the start of FORZETTO, a Phase 3 trial of zeleciment rostudirsen (z-rostudirsen, also called DYNE-251) in people with Duchenne muscular dystrophy who can benefit from exon 51 skipping. The 72-week study will enroll about 90 ambulatory males, and the first site is now open for enrollment.
The main goal of the trial is to measure rise from floor (RFF) velocity, a test of how quickly participants can stand up from the floor. Researchers will also look at several secondary endpoints tied to muscle and lung function. Dyne said the study design and protocol were aligned with the FDA, and the trial is meant to support potential U.S. traditional approval as well as marketing applications outside the U.S. The company said the design reflects input from the Duchenne community and a focus on meaningful functional improvement.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- March 8, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced additional positive data from the ongoing Phase 1/2 DELIVER clinical trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics reported additional positive data from the ongoing Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251) in people with Duchenne muscular dystrophy amenable to exon 51 skipping. New analyses out to 24 months showed improvement in heart and lung function compared with the expected declines seen in DMD natural history. Dyne says these findings build on earlier results showing sustained functional benefits across multiple clinical measures.
Dyne attributes the cardiopulmonary gains to its FORCE delivery platform, which is designed to reach a broad range of muscles including the heart, trunk, diaphragm, and the central nervous system. The analyses covered DELIVER participants who were randomized to z-rostudirsen at baseline and had cardiac magnetic resonance imaging and/or pulmonary function data available. The data will be presented in a poster titled “Zeleciment rostudirsen led to trends in long-term improvement in clinical outcomes including cardiopulmonary function: Additional data from DELIVER” (poster #476 LBT).
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- February 22, 2026
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that five presentations, including three oral presentations, highlighting the company's Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and Pompe disease programs will be presented at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 8-11, 2026, in Orlando, FL, and virtually.
AI Summary
Dyne Therapeutics announced five presentations — including three oral talks — at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference (March 8–11, Orlando and virtual) highlighting its programs in Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and Pompe disease.
Highlights include a late‑breaking poster showing new positive cardiopulmonary function trends from the DELIVER trial of z‑rostudirsen in DMD, and an oral presentation detailing the global Phase 3 trial design for z‑basivarsen in DM1 (Wednesday, March 11 at 9:30 a.m. ET; presenter Doug Kerr, M.D., Ph.D.). A second oral talk (Wednesday, March 11 at 11:15 a.m. ET) will present DYNE‑401 data for Pompe disease (presenter Tyler Picariello, Ph.D.). Each oral presentation will have a corresponding poster available during the conference poster sessions on March 9 and March 10.
Dyne says the five presentations across three neuromuscular diseases underscore the strength of its clinically validated FORCE™ platform; a symposium titled “Functional improvement: Moving beyond dystrophin in DMD” is set for March 9 at 7:00 a.m. ET.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- December 8, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
AI Summary
Dyne Therapeutics reported positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251) for people with Duchenne muscular dystrophy amenable to exon 51 skipping. The REC met its primary endpoint: muscle-content–adjusted dystrophin rose to a mean of 5.46% of normal at six months (p<0.0001), a roughly sevenfold increase from baseline at the registrational dose.
At six months, multiple prespecified functional measures showed improvement versus pooled placebo, with nominal statistical gains for Time to Rise velocity and 10-meter walk/run velocity. Forced vital capacity percent predicted (FVC%p) was preserved compared with a decline in placebo. Safety and tolerability remained favorable; most related adverse events were mild or moderate.
Long-term data from extension cohorts showed sustained functional benefits through 24 months. Dyne plans a U.S. regulatory submission in Q2 2026 to seek accelerated approval and is preparing for potential future launches and global studies.Read Announcement
- Drug:
- DYNE-251
- Announced Date:
- August 4, 2025
- Target Action Date:
- LATE 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc. announced that Data from the DELIVER registrational expansion cohort is expected in late 2025,
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- August 4, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, 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.
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- August 4, 2025
- Indication:
- Duchenne Muscular Dystrophy
Announcement
Dyne Therapeutics, Inc announced that potential BLA submission for U.S. accelerated approval anticipated in early 2026 -
Read Announcement- Drug:
- DYNE-251
- Announced Date:
- April 24, 2025
- Target Action Date:
- LATE 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 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:
- 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:
- 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:
- 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:
- 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
