# Dyne Therapeutics — Full Content > Consolidated, machine-readable content of Dyne Therapeutics' core website pages. > Source: https://www.dyne-tx.com/ > Compiled: July 2026; content verified against the live site on August 24, 2026 > Index file: https://www.dyne-tx.com/llms.txt > Scope: corporate, scientific, pipeline, clinical-trial, community and policy pages are reproduced in substance below. Patient stories are summarised, with links to the full narratives. Press releases and SEC filings live on https://investors.dyne-tx.com/ and are not duplicated here. Job postings are deliberately excluded. --- ## Company identity Legal name: Dyne Therapeutics, Inc. Also known as: Dyne Founded: 2017 Headquarters: 1560 Trapelo Road, Waltham, MA 02451, United States Phone: +1 781-786-8230 Fax: +1 781-786-8866 State of incorporation: Delaware, United States Stock exchange: Nasdaq Global Select Market Ticker symbol: DYN CUSIP: 26818M 108 Initial public offering: September 16, 2020 Fiscal year: January 1 – December 31 Employees: approximately 289 (as of August 2026) Company stage: clinical-stage biotechnology Tagline: "The muscle to keep life moving®" Transfer agent: Computershare Trust Company, N.A. Independent registered public accounting firm: Deloitte & Touche LLP, Boston, MA Outside legal counsel: Wilmer Cutler Pickering Hale and Dorr LLP, Boston, MA Contact addresses: - General inquiries: info@dyne-tx.com - Partnerships and business development: partnerships@dyne-tx.com - Patient advocacy: patientadvocacy@dyne-tx.com - Investors and media: ir@dyne-tx.com - Clinical trials: clinicaltrials@dyne-tx.com - Possible side effects of a Dyne investigational therapeutic: MedSafety@Dyne-tx.com (see Community guidelines below for the full reporting route) Web properties: - Corporate site: https://www.dyne-tx.com/ - Investor relations: https://investors.dyne-tx.com/ - U.S. healthcare professional site: https://dynemedicalcentral.com/ - LinkedIn: https://www.linkedin.com/company/dynetx - X (Twitter): https://x.com/dyne_tx - Facebook: https://www.facebook.com/DyneTherapeutic/ - YouTube: https://www.youtube.com/@dynetherapeutics --- ## Home Source: https://www.dyne-tx.com/ The muscle to keep life moving® Dyne Therapeutics is trailblazing the development of therapies to deliver functional improvement for people living with genetically driven neuromuscular diseases. **Pioneering approach.** Utilizing our FORCE™ platform to develop therapeutics that address the root cause of disease, delivering functional improvement for people living with neuromuscular disorders. **Pipeline.** Developing a broad portfolio of therapeutics for genetically driven neuromuscular diseases. **Find a Dyne clinical trial.** Conducting clinical trials for neuromuscular diseases. **Commitment.** Working to deliver functional improvement for people living with neuromuscular diseases. **Culture and careers.** Our commitment to people living with neuromuscular diseases is our greatest strength. --- ## About Us Source: https://www.dyne-tx.com/about-us/ Trailblazing the development of targeted therapies to deliver functional improvement. We are on a mission to deliver functional improvement for people living with genetically driven neuromuscular diseases through targeted therapies enabled by our FORCE™ platform. We have assembled an exceptional team with deep experience in neuromuscular disease, rare disorders, drug development, and manufacturing and commercialization of novel therapeutics. We have also established a scientific advisory board comprised of leading experts in neuromuscular disease drug discovery and development. ### Leadership team Our leadership team brings deep knowledge and extensive experience in the discovery, clinical development and commercialization of treatments for rare genetic diseases and novel therapeutic modalities. - John Cox, MBA — President and Chief Executive Officer - Doug Kerr, M.D., Ph.D., MBA — Chief Medical Officer - Erick J. Lucera — Chief Financial Officer - Oxana Beskrovnaya, Ph.D. — Chief Innovation Officer - Ranjan (Ron) Batra, Ph.D. — Chief Scientific Officer - Johanna Friedl-Naderer — Chief Commercial Officer - Monica Fay — Chief Medical Affairs Officer - Debra Feldman — Chief Regulatory Affairs Officer - Vikram (Vik) Ranade — Chief Business Officer - Raj Manchanda — Chief Technical Officer - James P. Bilotta, MBA — Chief Digital Information Officer - Lucia Celona — Chief Human Resources Officer - Alessandro Martuscelli — Chief Legal Officer - José Juves — Chief Corporate & Patient Affairs Officer - Axel Wiest — Senior Vice President, Head of Portfolio Strategy & Operations ### Board of directors - Jason Rhodes — Chairman - John Cox - Barry Greene - Ed Hurwitz - Carlo Incerti, M.D. - Vikram Karnani - Dirk Kersten - David Lubner - Brian Posner - Catherine Stehman-Breen ### Scientific advisory board - Sudhir Agrawal, D.Phil. - Nancy Andrews, M.D., Ph.D. - Louis Kunkel, Ph.D. - Francesco Muntoni, FRCPCH, FMedSci - Jeffrey Statland, M.D. - Charles Thornton, M.D. ### Contact and offices For general inquiries, email info@dyne-tx.com. To learn more about partnerships or working together, email partnerships@dyne-tx.com. To connect with the Patient Advocacy team, email patientadvocacy@dyne-tx.com. For investor and media inquiries, email ir@dyne-tx.com. Offices: 1560 Trapelo Road, Waltham, MA 02451. Phone: 781.786.8230. Fax: 781.786.8866. --- ## Our FORCE™ Platform Source: https://www.dyne-tx.com/our-forcetm-platform/ Advancing targeted therapeutics to address the root cause of disease. Dyne created the FORCE™ platform to develop therapeutics that address the root cause of disease and deliver functional improvement for people living with neuromuscular disorders. The FORCE platform consists of an antigen-binding fragment (Fab), a linker and a payload that is rationally designed to target the genetic basis of the disease we seek to treat. Dyne has clinical programs for Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), as well as preclinical programs for Pompe disease and multiple additional DMD mutations. Our proprietary Fab binds to transferrin receptor 1 (TfR1) to enable delivery to muscle and the central nervous system (CNS) with the goal of providing functional improvement for people living with neuromuscular diseases. We believe a Fab offers significant advantages over a full antibody, including enhanced tissue penetration, increased tolerability and reduced risk of immune system activation. Our Fab is linked to a therapeutic payload, such as an oligonucleotide or biologic, and the linker is chosen based on payload chemistry. The payload is carefully selected to directly address the root cause of the disease with the goal of delivering functional improvement. We believe our FORCE platform provides several potential advantages, including delivery to muscle and CNS, extended time between doses and ability to re-dose. Payload modalities used across the pipeline: antisense oligonucleotide (ASO), phosphorodiamidate morpholino oligomer (PMO), small interfering RNA (siRNA), and biologics. --- ## Pipeline Source: https://www.dyne-tx.com/pipeline/ A pipeline built to deliver functional improvement for neuromuscular diseases. We are advancing therapeutics to provide functional improvement for people living with genetically driven neuromuscular diseases. By utilizing our FORCE™ platform, we believe we will be able to deliver targeted therapies to muscle and the central nervous system (CNS) to address the root cause of disease. Dyne has a broad pipeline. The DMD, DM1 and FSHD programs are in clinical development; Pompe disease and multiple additional DMD exon-skipping candidates remain preclinical. Each of these neuromuscular diseases has a profound impact on affected communities around the world. ### Pipeline summary | Disease | Prevalence (U.S. / Europe) | Target | Candidate | Stage | |---|---|---|---|---| | Duchenne muscular dystrophy (DMD) | ~12,000 / ~16,000 | Exon 51 | Zeleciment rostudirsen (z-rostudirsen, DYNE-251) | Commercial column on Dyne's pipeline chart; remains investigational; BLA under Priority Review, PDUFA January 21, 2027 | | Myotonic dystrophy type 1 (DM1) | ~40,000 / ~55,000 | DMPK | Zeleciment basivarsen (z-basivarsen, DYNE-101) | Registration | | Facioscapulohumeral muscular dystrophy (FSHD) | ~15,000–40,000 / ~20,000–50,000 | DUX4 | DYNE-302 | Clinical — IND cleared July 28, 2026 | | Duchenne muscular dystrophy (DMD) | ~12,000 / ~16,000 | Exon 53 | DYNE-253 | Preclinical | | Duchenne muscular dystrophy (DMD) | ~12,000 / ~16,000 | Exon 45 | DYNE-245 | Preclinical | | Duchenne muscular dystrophy (DMD) | ~12,000 / ~16,000 | Exon 44 | DYNE-244 | Preclinical | | Duchenne muscular dystrophy (DMD) | ~12,000 / ~16,000 | Exon 55 | DYNE-255 | Preclinical | | Pompe disease | ~4,500 / ~5,500 | GAA | DYNE-401 | Preclinical | Pipeline expansion opportunities: CNS, rare skeletal, cardiac and metabolic muscle diseases. ### Zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) — DMD Z-rostudirsen is an investigational therapeutic for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. The registrational expansion cohort of the global Phase 1/2 DELIVER clinical trial of z-rostudirsen met its primary endpoint. Data from the DELIVER trial served as the basis for a Biologics License Application (BLA) for potential U.S. Accelerated Approval, which has been granted Priority Review. Z-rostudirsen continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial. Z-rostudirsen consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). It is designed to enable the production of near-full-length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement. It is administered intravenously every four weeks, as proposed in the submitted BLA. Regulatory designations: Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA); Orphan Drug designation from the FDA, the European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan, for the treatment of individuals with DMD amenable to exon 51 skipping. In addition to z-rostudirsen, Dyne is building a DMD franchise and has preclinical programs targeting other exons, including DYNE-253, DYNE-245, DYNE-244 and DYNE-255. ### About Duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) is a rare, X-linked and progressive neuromuscular disorder. DMD affects approximately 12,000 people in the U.S. and approximately 16,000 in the EU. Onset: DMD is the most common childhood-onset form of muscular dystrophy. Loss of strength and function typically first appear between 3 and 5 years of age. Cause: DMD results from mutations in the DMD gene which lead to total absence or nearly undetectable levels of dystrophin protein, essential for muscle structure, function and preservation. Symptoms may include: - Progressive muscle weakness that worsens over time, typically beginning in the hips, pelvis and upper legs before progressing to affect the shoulders, arms, trunk, neck and other muscle groups throughout the body - Cognitive function impairment and neuropsychiatric disorders (e.g., intellectual disability, learning disability, behavioral difficulties) - Enlarged calf muscles - Delays in developing motor skills such as sitting, standing or walking - Toe walking or waddling gait - Breathing problems - Cardiomyopathy, or inflammation of the muscles in the heart - Varying degrees of cognitive impairment in approximately one third of patients Unmet need: There is a significant unmet need for new treatment options that deliver functional improvement. ### Zeleciment basivarsen (z-basivarsen, also known as DYNE-101) — DM1 Z-basivarsen is an investigational therapeutic being evaluated in the fully enrolled global Phase 1/2 ACHIEVE clinical trial and the global confirmatory Phase 3 HARMONIA clinical trial for people living with DM1. Z-basivarsen consists of an antisense oligonucleotide (ASO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1) to enable delivery to muscle and the central nervous system. It is designed to deliver functional improvement in individuals living with DM1 by reducing toxic nuclear DMPK RNA to release splicing proteins and allow normal mRNA processing. Regulatory designations: Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA; Orphan Drug designation from the EMA and Japan's MHLW for the treatment of DM1. ### About myotonic dystrophy type 1 (DM1) Myotonic dystrophy type 1 (DM1) is a rare, progressive, genetic neuromuscular disease with high morbidity and early mortality. DM1 affects approximately 40,000 people in the U.S. and approximately 55,000 people in the EU. The severity of symptoms and rate of progression varies. Onset: Symptoms can begin at any point in an affected person's life, depending on the DM1 subtype. Adult-onset DM1 symptoms typically appear between 20 to 40 years of age. Cause: DM1 is caused by mutations in the DMPK gene, leading to a widespread disruption of RNA splicing, known as spliceopathy, which drives the multi-system manifestations of the disease. Symptoms include: - Muscle weakness throughout the body - Myotonia, or difficulty relaxing muscles - Cognitive impairments, excessive daytime sleepiness, fatigue and dysregulated sleep - Cardiac arrhythmias - Respiratory issues - Gastrointestinal dysfunction Unmet need: Although the genetic cause of DM1 is well understood, there are currently no approved disease-modifying treatments for DM1. ### DYNE-302 — FSHD DYNE-302 is Dyne's product candidate being developed for people living with FSHD. DYNE-302 consists of an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1), conjugated to an siRNA designed to reduce DUX4 expression. Dyne has generated extensive preclinical data that demonstrate robust and durable DUX4 suppression and functional improvement in a preclinical in vivo model developed by Dyne. On July 28, 2026, Dyne announced that the FDA had cleared the Investigational New Drug (IND) application for DYNE-302, allowing a Phase 1 clinical trial to begin. The pipeline chart lists DYNE-302 as clinical stage, IND cleared. It is Dyne's third program to enter clinical development using the FORCE platform, and Dyne intends to pursue a traditional approval pathway in the U.S. for DYNE-302. The trial design is described under Clinical Trials below. ### About facioscapulohumeral muscular dystrophy (FSHD) FSHD is a rare, progressive, genetic disease caused by a mutation in the DUX4 gene, leading to aberrant DUX4 expression in muscle with consequent damage and loss of function. These restrict daily activities and have a high physical, emotional and financial burden. FSHD affects approximately 15,000 to 40,000 people in the U.S. and approximately 20,000 to 50,000 people in the EU. Onset: There is wide variability in age of onset with FSHD. Symptoms typically begin to appear in the teen years or early adulthood. Cause: Individuals with FSHD carry a genetic mutation that allows the DUX4 gene to be sporadically activated in muscle cells, causing their gradual destruction throughout the body. Symptoms: The symptoms of FSHD often emerge first with a loss of facial muscle strength, making it difficult to smile or use a straw. Weakness typically progresses to all major muscle groups including the arms, torso, legs and abdomen, leading to limited mobility. Unmet need: Although the genetic cause of FSHD is well understood, there are currently no approved disease-modifying treatments for FSHD. ### Expansion opportunities We intend to utilize our FORCE platform to expand our portfolio by pursuing the development of programs in additional indications, including additional rare skeletal, CNS, cardiac and metabolic muscle diseases. By rationally selecting therapeutic payloads to conjugate with our proprietary antibody and linker, we believe we can develop product candidates to address the genetic basis of additional muscle diseases. --- ## Clinical Trials Source: https://www.dyne-tx.com/clinical-trials/ Clinical trials to advance our lead programs. Dyne Therapeutics is focused on delivering functional improvement for people living with genetically driven neuromuscular diseases. Clinical trials, a type of research that studies the safety and efficacy of potential therapeutics in people, must be conducted before bringing new medicines to patients. The U.S. Food and Drug Administration (FDA) and other global healthcare regulatory agencies review preclinical data on investigational medicines before clinical trials may start. Clinical trials would not be possible without the collaboration of the people living with the diseases we aim to treat, healthcare professionals and advocacy groups. ### HARMONIA (Phase 3, DM1) HARMONIA is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 clinical trial evaluating the efficacy, safety and tolerability of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in people living with myotonic dystrophy type 1 (DM1). The trial will enroll approximately 150 participants age 16 and older, randomized 1:1, who will receive 6.8 mg/kg of z-basivarsen or placebo once every eight weeks for 48 weeks. Participants who complete the placebo-controlled period may enter a long-term extension and receive 6.8 mg/kg of z-basivarsen every eight weeks for up to 24 additional weeks. Primary endpoint: change from baseline in the five times sit to stand (5xSTS) test at week 49. The 5xSTS test is a reliable and responsive measure that reflects key areas of DM1 impairment, including lower extremity strength, balance and trunk strength, which are critical to performing daily activities. Secondary endpoints include video hand opening time, quantitative muscle testing, the 10-meter walk/run test, the Myotonic Dystrophy Health Index, and additional patient- and clinician-reported outcomes. The trial also includes a broad set of exploratory endpoints designed to assess multiple domains of DM1 central nervous system impact. Study treatment and all study tests and procedures are provided at no cost to participants, who are reimbursed for approved travel-related expenses. Registry: ClinicalTrials.gov NCT07486934. ### ACHIEVE (Phase 1/2, DM1) ACHIEVE is a global, randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of zeleciment basivarsen (z-basivarsen, also known as DYNE-101) in patients with myotonic dystrophy type 1 (DM1). The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 6.8 mg/kg z-basivarsen administered every eight weeks. A registrational expansion cohort to support potential regulatory submissions, including Accelerated Approval in the U.S., is fully enrolled. Primary endpoint for the registrational expansion cohort: change from baseline in middle finger myotonia as measured by video hand opening time (vHOT) at 6 months, compared to placebo. Registries: ClinicalTrials.gov NCT05481879; EU Clinical Trials Information System EUCT 2023-510353-42-00. ### FORZETTO (Phase 3, DMD) FORZETTO is a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 clinical trial evaluating the efficacy, safety and tolerability of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in ambulatory male participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The trial will enroll approximately 90 participants 4 to 18 years of age, randomized 1:1, who will receive 20 mg/kg of z-rostudirsen or placebo once every four weeks for 72 weeks. Participants who complete the placebo-controlled period may enter a long-term extension and receive 20 mg/kg of z-rostudirsen once every four weeks for up to an additional 96 weeks. Primary endpoint: change from baseline in rise from floor (RFF) velocity, also referred to as time to rise (TTR), at week 73. RFF velocity is a clinically meaningful measure of muscle strength and motor coordination commonly assessed in DMD clinical trials. Secondary endpoints include changes from baseline in stride velocity 95th centile (SV95C), North Star Ambulatory Assessment (NSAA) total score, 10-meter walk/run (10MWR) velocity, four-stair climb (4SC) velocity and forced vital capacity percent predicted (FVC%p), as well as additional functional and patient-reported outcome measures. Dyne has aligned with the FDA on the design and protocol for FORZETTO. The trial is designed to confirm earlier findings and support the potential transition from Accelerated Approval to traditional approval in the United States, as well as to support regulatory submissions outside the U.S. Registries: ClinicalTrials.gov NCT07608432; EU Clinical Trials Information System EUCT 2025-524096-23-00. ### DELIVER (Phase 1/2, DMD) DELIVER is a global, randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial that evaluated the safety, tolerability and efficacy (as measured by both biomarker and functional improvement) of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg of z-rostudirsen administered every four weeks. The placebo-controlled portion of the registrational expansion cohort (REC) to support a potential regulatory submission for U.S. Accelerated Approval has been completed. Primary endpoint for the REC: change from baseline in dystrophin protein levels as measured by Western blot at 6 months. Participants from the MAD and REC portions had the option to enroll in the open-label extension and long-term extension portions of the study. Topline results announced December 8, 2025: the REC met its primary endpoint, demonstrating a statistically significant increase in dystrophin protein expression, and improvement relative to placebo was seen across all six prespecified functional endpoints at six months in a cohort of 32 participants, covering upper limb, lower limb and lung function. Sustained functional improvement from baseline was observed through 18 and 24 months in the MAD cohort. Registries: ClinicalTrials.gov NCT05524883; euclinicaltrials.eu EUCT 2023-510351-31-00. ### DYNE-302 Phase 1 (FSHD) On July 28, 2026, Dyne announced FDA clearance of the Investigational New Drug (IND) application for DYNE-302, allowing the start of a Phase 1 clinical trial in FSHD. The planned trial is a randomized, placebo-controlled, double-blind, multiple ascending dose (MAD) Phase 1 study in ambulatory adults ages 18 to 65 living with FSHD. The first group will enroll 9 participants who will receive three intravenous doses administered every four weeks, randomized 2:1 to DYNE-302 1.5 mg/kg (siRNA dose) or placebo. Following completion of this group, Dyne intends to evaluate higher doses and less frequent administration. Participants who complete the placebo-controlled period may enter an open-label long-term extension in which all participants receive DYNE-302 for up to an additional 96 weeks. Primary objective: safety and tolerability of DYNE-302. Additional objectives: pharmacokinetics and pharmacodynamics, and changes in DUX4-related biomarkers in muscle and blood, including muscle DUX4 transcriptome changes and plasma levels of the protein KHDC1L. Dyne intends to pursue a traditional approval pathway in the U.S. for DYNE-302. As of August 24, 2026 no registry number for this trial has been published, and the trial is not yet listed on the Clinical Trials page of dyne-tx.com. Clinical trial inquiries: clinicaltrials@dyne-tx.com. Patient advocacy: patientadvocacy@dyne-tx.com. ### Patient access and expanded access policy Expanded access (sometimes referred to as "managed access" or "early access") programs (EAP) refer to the use of an investigational medicine when the primary purpose is to diagnose, monitor, or treat a patient's disease rather than to obtain the kind of information about the drug that is generally derived from clinical trials. - EAPs are appropriate when substantial scientific evidence has been established to support both the safety and the efficacy of an investigational medicine in patients. - An investigational medicine addresses a specific disease and is not yet approved for commercial use or is only approved for testing in clinical trials for that disease by regulatory agencies, such as the FDA or EMA. Currently, Dyne does not have investigational medicines available through an expanded access program. Dyne will continue to assess this as its clinical programs progress. Dyne believes that participating in clinical trials is the best way for patients to access investigational medicines. These carefully controlled, rigorous scientific studies are needed to obtain regulatory approval, which will be critical for enabling the broadest access to treatment for a specific condition. - Dyne will strive to design trials that are inclusive of as many patients as possible, but recognizes that not every patient will be eligible for these trials. - For those patients who have completed a Dyne clinical trial, it is Dyne's plan, when supported by the scientific data, to provide access to the investigational medicine until it is approved and available locally. Dyne conducts regular review of its policies to ensure conformity with applicable laws and regulations, and reserves the right to revise this policy at any time. --- ## Our Commitment Source: https://www.dyne-tx.com/our-commitment/ Committed to the communities we serve. At Dyne, we are committed to listening to and learning from the communities that we aim to serve, including individuals and families living with Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). Their stories inspire us. Equally important, these insights guide our development of targeted therapies designed to deliver what patients value most: functional improvement. ### Letters to our communities - Dyne Receives FDA Clearance to Start Early-Stage FSHD Clinical Trial (July 28, 2026) - Together in Progress: FDA Accepts BLA for Z-Rostudirsen and Grants Priority Review (July 20, 2026) - Zeleciment Rostudirsen Application Submitted for U.S. Accelerated Approval; Phase 3 Confirmatory FORZETTO Trial Initiated (May 26, 2026) - New Clinical Trial Opportunity in DM1: HARMONIA Phase 3 Study Evaluating Zeleciment Basivarsen (April 16, 2026) - Historic Step Forward: Duchenne Added to Newborn Screening (December 16, 2025) - Dyne Therapeutics announces positive topline results from Phase 1/2 DELIVER trial of z-rostudirsen in Duchenne muscular dystrophy (DMD) (December 8, 2025) - Dyne receives FDA Breakthrough Therapy Designation for DYNE-251 for the treatment of patients with DMD (August 4, 2025) The content of each letter is set out in the section "Letters to the community" below. ### Awareness days Rare Disease Day: Rare Disease Day aims to raise awareness of the impact that rare diseases have on more than 300 million people around the world. In honor of Rare Disease Day, Dyne hosted members of the neuromuscular disease community to reflect on the 2026 theme, "More Than You Can Imagine." Dyne has marked Rare Disease Day annually since 2020. World FSHD Day (June 20): Dyne joins the FSHD community in recognizing World FSHD Day. Dynamos share orange slice selfies to support the FSHD Society's annual campaign to build awareness of facial weakness experienced by some people living with FSHD that can make smiling difficult. World Duchenne Awareness Day (September 7): Dyne joins the Duchenne community in recognizing World Duchenne Awareness Day. International Myotonic Dystrophy Awareness Day (September 15): Dyne marks International Myotonic Dystrophy Awareness Day annually. --- ## Letters to the community Dyne publishes letters to the DMD, DM1 and FSHD communities at milestone moments. Each letter is signed "The Dyne Therapeutics Team". All product candidates named below are investigational and have not been approved by the FDA or any other regulatory authority for commercial use; their safety and efficacy have not been established. ### Dyne Receives FDA Clearance to Start Early-Stage FSHD Clinical Trial July 28, 2026 — https://www.dyne-tx.com/early-stage-fshd-clinical-trial/ Addressed to the FSHD community. Dyne announced that it had received clearance from the FDA for its Investigational New Drug (IND) application to initiate a Phase 1 clinical trial of DYNE-302 in facioscapulohumeral muscular dystrophy, allowing the company to begin evaluating DYNE-302 in people living with FSHD. DYNE-302 is designed to reduce expression of DUX4, a protein believed to be the underlying cause of FSHD, and leverages the FORCE™ platform used in Dyne's clinical-stage DMD and DM1 programs. Planned Phase 1 design: randomized, placebo-controlled, double-blind, multiple ascending dose trial in ambulatory adults ages 18–65 living with FSHD. The primary objective is safety and tolerability; additional objectives cover pharmacokinetics, pharmacodynamics and DUX4-related biomarkers in muscle and blood, including the blood protein KHDC1L. The first group of 9 participants receives three intravenous doses every four weeks, randomized 2:1 to DYNE-302 1.5 mg/kg or placebo; higher doses and less frequent administration are to be evaluated afterwards. Participants completing the placebo-controlled period may enter an open-label long-term extension of up to 96 additional weeks. Dyne intends to pursue a traditional approval pathway in the U.S. ### Together in Progress: FDA Accepts BLA for Z-Rostudirsen and Grants Priority Review July 20, 2026 — https://www.dyne-tx.com/fda-accepts-bla-for-z-rostudirsen/ Addressed to the Duchenne community. The FDA accepted for review the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) for individuals living with DMD amenable to exon 51 skipping, granted the application Priority Review and assigned a PDUFA target action date of January 21, 2027. Acceptance of a BLA means the FDA has determined the application is sufficiently complete to undergo review; it does not guarantee approval. Assuming approval on the anticipated timeline, Dyne continues to expect a potential U.S. launch of z-rostudirsen in the first quarter of 2027. Key points from the letter: the BLA includes data from the positive Phase 1/2 DELIVER trial and seeks Accelerated Approval; Priority Review reduces the review process from 10 months to 6 months; z-rostudirsen continues to be evaluated in the DELIVER long-term extension and in the Phase 3 FORZETTO trial; the candidate is designed to enable production of near-full-length dystrophin in muscle and CNS; it is administered intravenously every four weeks as proposed in the BLA. The letter also names four additional development candidates — DYNE-253, DYNE-245, DYNE-244 and DYNE-255 — for DMD amenable to exon 53, 45, 44 and 55 skipping respectively. Contact for additional information: patientadvocacy@dyne-tx.com. ### Zeleciment Rostudirsen Application Submitted for U.S. Accelerated Approval; Phase 3 Confirmatory FORZETTO Trial Initiated May 26, 2026 — https://www.dyne-tx.com/forzetto-trial-initiated/ Addressed to the Duchenne community. Dyne announced submission of the BLA to the FDA for z-rostudirsen for DMD amenable to exon 51 skipping, based on data from the positive Phase 1/2 DELIVER trial, seeking Accelerated Approval and requesting Priority Review. The letter also reported the initiation of the Phase 3 confirmatory FORZETTO trial, announced the previous week. FORZETTO design as described in the letter: global, randomized, placebo-controlled, double-blind confirmatory Phase 3 trial of intravenous z-rostudirsen in ambulatory male participants with DMD amenable to exon 51 skipping; approximately 90 participants aged 4 to 18, randomized 1:1 to z-rostudirsen 20 mg/kg or placebo every four weeks; the first U.S. trial site was activated and open to enrollment, with additional U.S. and then ex-U.S. sites to follow; primary endpoint is change from baseline in rise from floor velocity (time to rise) at week 73; secondary endpoints include SV95C, NSAA total score, 10MWR velocity, four-stair climb velocity and FVC%p, plus additional functional and patient-reported outcomes; a 96-week open-label long-term extension follows the 72-week double-blind period. Dyne has aligned with the FDA on the FORZETTO design and protocol. Trial inquiries: clinicaltrials@dyne-tx.com. ### New Clinical Trial Opportunity in DM1: HARMONIA Phase 3 Study Evaluating Zeleciment Basivarsen April 16, 2026 — https://www.dyne-tx.com/new-clinical-trial-opportunity-in-dm1/ Addressed to the myotonic dystrophy community. With two DM1 trials ongoing, Dyne shared information about opportunities to participate in studies of zeleciment basivarsen (z-basivarsen, also known as DYNE-101). HARMONIA, the global Phase 3 trial, was announced in March 2026 at the Muscular Dystrophy Association Conference and is listed on ClinicalTrials.gov; additional sites inside and outside the United States were expected to open over the following months. Enrollment in the Phase 1/2 ACHIEVE trial continues. HARMONIA design as described in the letter: approximately 150 participants age 16 and older, randomized 1:1 to 6.8 mg/kg z-basivarsen or placebo every eight weeks; the primary goal is change from baseline in the five times sit to stand (5xSTS) test at week 49; additional assessments include video hand opening time, strength testing, the 10-meter walk/run test, the Myotonic Dystrophy Health Index and other patient- and clinician-reported outcomes, plus a broad set of exploratory endpoints covering DM1 CNS impact; a 24-week long-term extension follows the 48-week double-blind period. Study treatment, tests and procedures are provided at no cost, approved travel expenses are reimbursed, and travel support services may be available. Trial inquiries: clinicaltrials@dyne-tx.com. ### A Historic Step Forward: Duchenne Added to Newborn Screening December 16, 2025 — https://www.dyne-tx.com/community-letter-duchenne-added-to-newborn-screening/ Dyne congratulated Parent Project Muscular Dystrophy (PPMD), the Muscular Dystrophy Association (MDA), the U.S. Department of Health and Human Services (HHS), the Health Resources and Services Administration (HRSA), partner organizations, allies and families on the addition of Duchenne muscular dystrophy to the Recommended Uniform Screening Panel (RUSP). With Duchenne included in newborn screening, more families will have the opportunity to receive an earlier diagnosis, potentially accelerating and expanding access to clinical care and therapies. ### Positive Topline Results from the Phase 1/2 DELIVER Trial of Z-Rostudirsen in DMD December 8, 2025 — https://www.dyne-tx.com/community-letter-deliver-clinical-trial/ Addressed to the Duchenne community. Dyne announced positive topline results from the DELIVER trial along with long-term data showing sustained benefit out to 24 months. DELIVER consists of Part 1, a multiple ascending dose (MAD) study to identify the appropriate dose, and Part 2, the Registrational Expansion Cohort (REC) testing the selected dose in a larger number of participants; participants who completed MAD and REC continue to receive z-rostudirsen in the open-label extension. Results reported: the REC met its primary endpoint, demonstrating a statistically significant increase in dystrophin protein expression; improvement relative to placebo was seen across all six prespecified endpoints at six months in the 32-participant cohort, covering upper limb, lower limb and lung function; sustained functional improvement from baseline was observed through 18 and 24 months in each of the six endpoints for the MAD cohort. Safety, based on participants followed for up to 36 months (data as of August 19, 2025): z-rostudirsen continued to demonstrate a favorable safety profile, with most related treatment-emergent adverse events mild or moderate and no related serious TEAEs in the REC. Since the previous safety update, two participants in the open-label portions experienced malaise and/or pyrexia reported as related serious TEAEs; both fully recovered and continued to receive z-rostudirsen without interruption. Next steps stated at the time: BLA submission for U.S. Accelerated Approval in Q2 2026; initiation of a global Phase 3 trial in Q2 2026; potential U.S. launch in Q1 2027 assuming the FDA grants Priority Review; continued pursuit of approval pathways outside the U.S. ### To the Duchenne Muscular Dystrophy Community — FDA grants Breakthrough Therapy Designation to DYNE-251 August 4, 2025 — https://www.dyne-tx.com/community-letter-duchenne-muscular-dystrophy-community/ Dyne announced that the FDA had granted Breakthrough Therapy Designation to DYNE-251 for the treatment of patients with DMD amenable to exon 51 skipping. Breakthrough Therapy Designation is granted to therapies that show early promise of being significantly better than current treatments in improving meaningful outcomes for patients, and brings added support from senior FDA experts along with early and frequent communication. The letter noted that data from the ongoing DELIVER Registrational Expansion Cohort were expected in late 2025 and were expected to support a potential submission for Accelerated Approval in the United States in early 2026. Note: on the Our Commitment page this letter is listed under the headline "Dyne receives FDA Breakthrough Therapy Designation for DYNE-251…", while the page itself is titled "To the Duchenne Muscular Dystrophy Community". --- ## Culture + Careers Source: https://www.dyne-tx.com/careers-culture/ Our commitment to people with neuromuscular diseases is our greatest strength. We are fearless innovators on a mission to deliver functional improvement for individuals, families and communities. We pursue our goals with passion, determination, agility and urgency, recognizing that countless lives depend on our efforts. ### Core values - **Delivering for patients** — Our purpose: functional improvement for people living with neuromuscular diseases. - **One Dyne community** — Our commitment: thrive through resiliency, transparency, respect, inclusion and honesty. - **One mission, no ego** — Our vision: united in purpose, selfless in action. - **Embrace accountability & excellence** — Our pledge: own the enterprise outcome, deliver with excellence. - **Fearless innovation** — Our resolve: think bigger, act bolder, aim higher and never fear failure. Dyne employees are referred to as "Dynamos." --- ## Corporate Responsibility Source: https://www.dyne-tx.com/corporate-responsibility/ Dyne Therapeutics is a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases by addressing the root cause of disease through its FORCE™ platform. The platform uses an antigen-binding fragment (Fab) that leverages transferrin receptor 1 (TfR1) to enable broad distribution to muscle and the central nervous system (CNS). Corporate responsibility pillars: 1. Exceptional team fostering a differentiated culture 2. Delivering for patients and community 3. Protecting the environment 4. Corporate governance and operating the business with integrity As of August 2026, Dyne employed approximately 289 Dynamos. In 2025, Dyne offered more than 14 different development sessions and a management development journey totaling over 200 hours of formal development opportunities, covering manager effectiveness, communication, accountability, feedback, presentation development and delivery, and time and meeting management. Dyne holds quarterly performance and development check-in conversations. --- ## Community guidelines Source: https://www.dyne-tx.com/community-guidelines/ Dyne's guidelines for its corporate social media channels on X, LinkedIn and Facebook. Because Dyne operates in a regulated industry, all of its social media engagement must adhere to the ethical, legal and regulatory standards for the biopharmaceutical industry, so engagement may at times seem limited or delayed. - Keep the conversation on topics related to Dyne or the communities it serves; no unsupported accusations or claims, misleading information, promotion of goods or services, or spam. - Dyne reserves the right to remove content and to accept, reject or block users at any time. - Respect one another: no foul language, disparagement or vulgar images. - Protect privacy: do not post confidential or identifying information about yourself or anyone else. - Because of the legal requirements Dyne operates under, it does not offer medical advice or discuss specific products or treatment options, its own or other companies'. Anyone experiencing a medical problem should consult a healthcare professional. Reporting a possible side effect (the page's stated safety policy): anyone who believes they have experienced a possible side effect related to a Dyne investigational therapeutic should contact their healthcare provider immediately. Possible side effects are encouraged to be reported to MedSafety@Dyne-tx.com. Reports may also be made directly to the FDA at www.fda.gov/medwatch or by calling 1-800-FDA-1088, or to the local health authority outside the U.S. Information posted publicly about a possible side effect may be removed, and Dyne may contact the person for additional information in order to comply with regulatory guidelines. Dyne claims no responsibility for the accuracy of content on sites it links to, and following or interacting with third-party accounts is not an indication of endorsement. In addition to the privacy policy and terms of use of each third-party social media platform, use of Dyne's corporate social properties is governed by Dyne's Privacy Policy. --- ## Scientific Publications & Presentations Source: https://www.dyne-tx.com/our-forcetm-publications/ Selected recent publications and presentations: - **July 7–11, 2026** — 19th International Congress on Neuromuscular Diseases (ICNMD): "FORZETTO, a Phase 3 Study of Z-Rostudirsen in Ambulatory Males with Exon 51 Amenable DMD" - **May 11–15, 2026** — American Society of Gene & Cell Therapy (ASGCT) Annual Meeting: "Targeting Tauopathies: Robust and Widespread MAPT Silencing in CNS of Mice and NHP with TfR1-mediated Oligonucleotide Delivery" - **March 22–25, 2026** — Society of Toxicology: "The FORCE™ Platform Does Not Cause Anemia in Repeat-Dose Preclinical Toxicity Studies" - **March 8–11, 2026** — Muscular Dystrophy Association (MDA) Clinical & Scientific Conference: multiple presentations, including "Zeleciment Rostudirsen Significantly Increased Dystrophin Protein Levels and Led to Functional Improvement in Clinical Measures in the DELIVER Trial"; "A Global Phase 3 Trial Assessing the Efficacy and Safety of Z-Basivarsen in Myotonic Dystrophy Type 1"; "DYNE-401 Demonstrates Potential to Address Pompe Disease with Low and Infrequent Dosing"; "Zeleciment Basivarsen Targets the Underlying Cause of DM1 to Enable Functional Improvement in the Phase 1/2 ACHIEVE Trial" - **February 28, 2026** — Journal of Medical Economics: "Clinical and Healthcare Burden of Myotonic Dystrophy Type 1 (DM1) in the United States: A Claims-Based Study" - **February 23, 2026** — Nucleic Acid Insights: "Reaching the CNS with Therapeutic Oligonucleotides via TfR1: Navigating Delivery and Distribution Challenges" - **October 19–21, 2025** — NORD Breakthrough Summit: "Health Insurance Literacy in Myotonic Dystrophy Type 1 (DM1), an Interim Report" - **October 7–11, 2025** — 30th Annual International Congress of the World Muscle Society: multiple presentations on DYNE-101 in ACHIEVE, DYNE-251 in DELIVER, and TfR1-mediated CNS delivery of exon-skipping PMO - **August 14, 2025** — Journal of Neuromuscular Diseases: "Changes in RNA splicing as a surrogate endpoint for myotonic dystrophy Type 1 (DM1) clinical trials" - **June 12–13, 2025** — FSHD International Research Conference: "DYNE-302 Leads to Functional Improvement and Resolves Muscle Transcriptomic Changes in Mouse Models of FSHD" Full list, including approximately 60 posters and presentations published as PDFs: https://www.dyne-tx.com/our-forcetm-publications/ --- ## Patient community resources Source: https://www.dyne-tx.com/resources/ Dyne maintains a curated list of patient advocacy organizations and information sources. Dyne assumes no responsibility for information, statements or other content encountered outside of Dyne's website. **DM1 — U.S.** - Myotonic Dystrophy Foundation — https://www.myotonic.org/ - Muscular Dystrophy Association — https://www.mda.org/ - National Organization for Rare Disorders — https://rarediseases.org/ - National Institutes of Health, Myotonic Dystrophy — https://rarediseases.info.nih.gov/diseases/10419/myotonic-dystrophy **DM1 — outside the U.S.** - Cure Myotonic Dystrophy UK — https://congenitalmyotonicdystrophy.org/ - Myotonic Dystrophy Support Group (UK) — https://www.myotonicdystrophysupportgroup.org/ - Treat-NMD (UK) — https://treat-nmd.org/ - Euro-Dyma (Europe) — https://euro-dyma.eu/ - Muscular Dystrophy UK — https://www.musculardystrophyuk.org/ - Myotonic Dystrophy Patients' Group of Japan — https://dm-family.net/ **DMD — U.S.** - Charley's Fund — https://charleysfund.org/ - CureDuchenne — https://www.cureduchenne.org/ - Jett Foundation — https://www.jettfoundation.org/ - Parent Project Muscular Dystrophy — https://www.parentprojectmd.org/ - Muscular Dystrophy Association — https://www.mda.org/ - National Institutes of Health, Duchenne Muscular Dystrophy — https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy - National Organization for Rare Disorders — https://rarediseases.org/ **DMD — outside the U.S.** - Defeat Duchenne Canada — https://defeatduchenne.ca/ - Action Duchenne (UK) — https://www.actionduchenne.org/ - French Muscular Dystrophy Association (AFM) — https://www.afm-telethon.com/ - Duchenne UK — https://www.duchenneuk.org/ - Muscular Dystrophy UK — https://www.musculardystrophyuk.org/ - Treat-NMD (UK) — https://treat-nmd.org/ - Parent Project Italy — https://parentproject.it/ - Progena Swiss Duchenne Foundation — https://progena.ch/ **FSHD — U.S.** - FSHD Society — https://www.fshdsociety.org/ - Friends of FSH Research — https://www.fshfriends.org/ - Chris Carrino Foundation for FSHD — https://chriscarrinofoundation.org/ - Muscular Dystrophy Association — https://www.mda.org/ - National Organization for Rare Disorders — https://rarediseases.org/ **FSHD — outside the U.S.** - Amis FSH (France) — https://www.amisfsh.fr/ - Treat-NMD (UK) — https://treat-nmd.org/ - French Muscular Dystrophy Association (AFM) — https://www.afm-telethon.com/ - Muscular Dystrophy UK — https://www.musculardystrophyuk.org/ - FSHD Europe — https://fshd-europe.info/ - FSHD Canada Foundation — https://fshd.ca/ --- ## Patient stories Dyne publishes first-person accounts from community members on three hub pages. Written stories are summarised here, with links to the full narratives; several accounts are published as video only. **DM1 Stories** — https://www.dyne-tx.com/dm1/ Living with DM1 has profound impacts on every part of people's lives, including work, social interactions and family relationships. Community members have shared their perspectives with Dyne, including what a potentially transformative therapeutic would mean to them. Featured: Sarah (video); BillyDean and Suzette (written story); Joachim and Tim (video); Joachim (written story); Loraine (written story). **DMD Stories** — https://www.dyne-tx.com/dmd/ Featured: Catherine and Alan (video); Diana and Jordan (video); Ravi (written story). **FSHD Stories** — https://www.dyne-tx.com/fshd/ Featured: Laura and Chelsea (video and written story); Rachel (video); Ian (video); Sam (video). Written stories: - **Meet BillyDean and Suzette (DM1)** — https://www.dyne-tx.com/dm1-story-billydean/ — BillyDean and Suzette battled the effects of myotonic dystrophy type 1 before they even knew its name. Through their resilience and Suzette's advocacy for her son, they succeeded in getting a DM1 diagnosis and have been learning how to live and thrive ever since. - **Meet Joachim (DM1)** — https://www.dyne-tx.com/dm1-story-joachim/ — "I'm not alone in this": one person's journey with DM1. Growing up, Joachim says, myotonic dystrophy "was always in my background and in the back of my head." - **Meet Loraine (DM1)** — https://www.dyne-tx.com/dm1-story-loraine/ — A courageous family battle and devotion in the face of generational adversity. - **Meet Ravi (DMD)** — https://www.dyne-tx.com/dmd-story-ravi/ — "I want to be someone": Ravi's journey with DMD. A law school student living with DMD, whose symptoms were first noticed by his uncle at age seven, Ravi studied biology and political science, works on environmental and disability advocacy, and joined the Parent Project Muscular Dystrophy Adult Advisory Committee to represent adults living with Duchenne. Interview conducted July 2020. - **Meet Laura and Chelsea (FSHD)** — https://www.dyne-tx.com/fshd-story-chelsea/ — A mother and daughter's joint perspective on their journey with FSHD. --- ## Privacy policy Source: https://www.dyne-tx.com/privacy-policy/ Summary of Dyne's website privacy policy. - Visitors may browse dyne-tx.com without providing any personal information. If a visitor contacts Dyne and provides personally identifiable information such as name, address, telephone number or email address, Dyne uses it for the purpose for which it was submitted and may share it with partners, consultants and service providers for that purpose. - Dyne does not send unsolicited messages to users who indicate they do not wish to receive them. To opt out, contact info@dyne-tx.com. - The site may collect domain information automatically to understand how the site is used; no personally identifiable information is gathered in that process. - The site may use cookies for registration and customisation. Browsers can be set to refuse cookies, though some pages may then not function fully. - Data may be stored in an electronic database by Dyne or by third parties working with it, and may be exchanged over the internet or intranet systems. Dyne may release personally identifiable data in response to a court order, subpoena, search warrant or applicable law, and states that it cannot guarantee that transmission over the internet is fully secure. - The website is not intended for, or designed to attract, children under the age of 13. - To stop receiving communications, or to request deletion of submitted information, contact info@dyne-tx.com. Dyne reserves the right not to remove or amend information regarding an adverse drug event or other information it is required to retain by law or regulation. - The site links to third-party websites as a convenience; those sites' own terms of use apply. --- ## Terminology glossary - **FORCE™ platform** — Dyne's proprietary delivery platform: an antigen-binding fragment (Fab) plus a linker plus a therapeutic payload. - **Fab** — antigen-binding fragment; a portion of an antibody. Dyne's Fab binds transferrin receptor 1. - **TfR1** — transferrin receptor 1; the receptor Dyne's Fab targets to enable delivery to muscle and CNS. - **ASO** — antisense oligonucleotide. - **PMO** — phosphorodiamidate morpholino oligomer. - **siRNA** — small interfering RNA. - **DUX4** — the gene whose aberrant expression in muscle causes FSHD; the target of DYNE-302. - **KHDC1L** — a blood protein used as a DUX4-related biomarker in the DYNE-302 Phase 1 trial. - **CNS** — central nervous system. - **DMD** — Duchenne muscular dystrophy. - **DM1** — myotonic dystrophy type 1. - **FSHD** — facioscapulohumeral muscular dystrophy. - **IND** — Investigational New Drug application; FDA clearance of an IND allows clinical trials to begin. - **BLA** — Biologics License Application; the submission seeking FDA approval of a biologic. - **PDUFA date** — the target action date by which the FDA aims to complete its review of an application. - **Dynamos** — Dyne employees. - **Functional improvement** — Dyne's stated development goal: measurable improvement in patients' physical function, not solely biomarker change.