# Dyne Therapeutics

> Dyne Therapeutics, Inc. (Nasdaq: DYN) is a clinical-stage biotechnology company headquartered in Waltham, Massachusetts, developing targeted therapeutics for genetically driven neuromuscular diseases. Dyne's proprietary FORCE™ platform conjugates an antigen-binding fragment (Fab) that binds transferrin receptor 1 (TfR1) to a therapeutic payload — such as an antisense oligonucleotide (ASO), phosphorodiamidate morpholino oligomer (PMO), siRNA or biologic — to enable delivery to muscle and the central nervous system (CNS) and address the root cause of disease. Dyne is advancing clinical programs in Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), and preclinical programs in Pompe disease and additional DMD exon-skipping targets.

Last updated: August 24, 2026. Consolidated content of the pages listed below, in a single file: https://www.dyne-tx.com/llms-full.txt

Key facts:

- Legal name: Dyne Therapeutics, Inc.
- Founded: 2017
- Headquarters: 1560 Trapelo Road, Waltham, MA 02451, USA
- Phone: +1 781-786-8230
- Publicly traded: Nasdaq Global Select Market, ticker DYN; IPO September 16, 2020; incorporated in Delaware
- Employees: approximately 289 (as of August 2026)
- Stage: clinical-stage biotechnology
- Core technology: FORCE™ platform (Fab–linker–payload conjugate targeting TfR1)
- Therapeutic areas: DMD, DM1, FSHD, Pompe disease
- Lead candidates: zeleciment rostudirsen (z-rostudirsen, DYNE-251) for DMD exon 51; zeleciment basivarsen (z-basivarsen, DYNE-101) for DM1
- Regulatory status: BLA for z-rostudirsen accepted by the FDA with Priority Review; PDUFA target action date January 21, 2027; potential U.S. launch expected in the first quarter of 2027 if approval is received on the anticipated timeline
- General inquiries: info@dyne-tx.com | Partnerships: 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 an investigational therapeutic: MedSafety@Dyne-tx.com

Diseases Dyne is targeting:

- Duchenne muscular dystrophy (DMD): rare, X-linked, progressive neuromuscular disorder caused by mutations in the DMD gene leading to absent or nearly undetectable dystrophin. Affects approximately 12,000 people in the U.S. and 16,000 in Europe. Onset typically between 3 and 5 years of age.
- Myotonic dystrophy type 1 (DM1): rare, progressive genetic neuromuscular disease caused by mutations in the DMPK gene that cause widespread disruption of RNA splicing (spliceopathy). Affects approximately 40,000 people in the U.S. and 55,000 in Europe. No approved disease-modifying treatments.
- Facioscapulohumeral muscular dystrophy (FSHD): rare, progressive genetic disease caused by aberrant DUX4 expression in muscle. Affects approximately 15,000–40,000 people in the U.S. and 20,000–50,000 in Europe. No approved disease-modifying treatments.
- Pompe disease: rare metabolic muscle disease associated with the GAA gene. Affects approximately 4,500 people in the U.S. and 5,500 in Europe.

## Company

- [Home](https://www.dyne-tx.com/): Company overview, mission and latest news.
- [About Us](https://www.dyne-tx.com/about-us/): Mission, leadership team, board of directors, scientific advisory board, offices and contact details.
- [Corporate Responsibility](https://www.dyne-tx.com/corporate-responsibility/): Workforce and culture initiatives, patient and community commitments, environmental practices, corporate governance and business integrity.
- [Culture + Careers](https://www.dyne-tx.com/careers-culture/): Core values, employee ("Dynamo") perspectives and benefits.
- [Investors & Media](https://investors.dyne-tx.com/): Press releases, SEC filings, stock information, corporate governance documents and investor resources.
- [Community Guidelines](https://www.dyne-tx.com/community-guidelines/): Rules for engagement on Dyne's corporate social media channels, and Dyne's stated route for reporting a possible side effect of an investigational therapeutic — contact a healthcare provider immediately, report to MedSafety@Dyne-tx.com, and optionally to the FDA at www.fda.gov/medwatch or 1-800-FDA-1088.
- [Privacy Policy](https://www.dyne-tx.com/privacy-policy/): How Dyne handles information collected through its website.

## Science and platform

- [Our FORCE™ Platform](https://www.dyne-tx.com/our-forcetm-platform/): How the FORCE platform works — Fab binding to TfR1, linker chemistry, oligonucleotide and biologic payloads, and the resulting delivery to muscle and CNS.
- [Scientific Publications & Presentations](https://www.dyne-tx.com/our-forcetm-publications/): Peer-reviewed publications, congress abstracts, posters and oral presentations covering the FORCE platform and Dyne's clinical and preclinical programs.

## Pipeline and programs

- [Pipeline](https://www.dyne-tx.com/pipeline/): Full portfolio across DMD, DM1, FSHD and Pompe disease, with disease prevalence, molecular targets, development stage and program descriptions.
- [Zeleciment rostudirsen (z-rostudirsen, DYNE-251) — DMD exon 51](https://www.dyne-tx.com/pipeline/#DMD): PMO–Fab conjugate designed to produce near-full-length dystrophin in muscle and CNS; administered intravenously every four weeks. Breakthrough Therapy, Fast Track and Rare Pediatric Disease designations from the FDA; Orphan Drug designation from FDA, EMA and Japan's MHLW. BLA under FDA Priority Review.
- [Zeleciment basivarsen (z-basivarsen, DYNE-101) — DM1](https://www.dyne-tx.com/pipeline/#DM1): ASO–Fab conjugate designed to reduce toxic nuclear DMPK RNA and restore normal mRNA processing. Breakthrough Therapy, Fast Track and Orphan Drug designations from the FDA; Orphan Drug designation from EMA and Japan's MHLW.
- [DYNE-302 — FSHD](https://www.dyne-tx.com/pipeline/#FSHD): siRNA–Fab conjugate designed to reduce DUX4 expression. Clinical stage — the FDA cleared the Investigational New Drug (IND) application on July 28, 2026, allowing a Phase 1 trial to begin. Dyne's third program to enter clinical development.
- [Preclinical DMD exon-skipping programs](https://www.dyne-tx.com/pipeline/#DMD): DYNE-253 (exon 53), DYNE-245 (exon 45), DYNE-244 (exon 44), DYNE-255 (exon 55).
- [Preclinical Pompe disease program](https://www.dyne-tx.com/pipeline/): DYNE-401, targeting GAA.

## Clinical trials

- [Clinical Trials](https://www.dyne-tx.com/clinical-trials/): Overview of active Dyne studies, trial design summaries and Dyne's patient access and expanded access policy.
- [HARMONIA — Phase 3, DM1](https://clinicaltrials.gov/study/NCT07486934): Global, randomized, placebo-controlled, double-blind confirmatory trial of z-basivarsen; approximately 150 participants aged 16 and older randomized 1:1; primary endpoint is change from baseline in the five-times sit-to-stand (5xSTS) test at week 49.
- [ACHIEVE — Phase 1/2, DM1](https://clinicaltrials.gov/study/NCT05481879): Global, randomized, placebo-controlled, double-blind trial of z-basivarsen; registrational expansion cohort fully enrolled; primary endpoint is change from baseline in video hand opening time (vHOT) at 6 months.
- [FORZETTO — Phase 3, DMD](https://clinicaltrials.gov/study/NCT07608432): Global, randomized, placebo-controlled, double-blind confirmatory trial of z-rostudirsen in approximately 90 ambulatory male participants aged 4 to 18 amenable to exon 51 skipping, randomized 1:1; primary endpoint is change from baseline in rise-from-floor velocity at week 73.
- [DELIVER — Phase 1/2, DMD](https://clinicaltrials.gov/study/NCT05524883): Global, randomized, placebo-controlled, double-blind trial of z-rostudirsen; registrational expansion cohort met its primary endpoint of change from baseline in dystrophin protein levels by Western blot at 6 months; long-term extension ongoing.
- [DYNE-302 Phase 1 — FSHD](https://www.dyne-tx.com/early-stage-fshd-clinical-trial/): Randomized, placebo-controlled, double-blind, multiple ascending dose trial in ambulatory adults aged 18 to 65; IND cleared by the FDA on July 28, 2026. No registry number published as of August 24, 2026.

## Patient and community

- [Our Commitment](https://www.dyne-tx.com/our-commitment/): Dyne's engagement with the DMD, DM1 and FSHD communities, letters to patient advocacy organizations, and Rare Disease Day, World FSHD Day, World Duchenne Awareness Day and International Myotonic Dystrophy Awareness Day activities.
- [DM1 Stories](https://www.dyne-tx.com/dm1/): First-person accounts from people and families living with myotonic dystrophy type 1.
- [DMD Stories](https://www.dyne-tx.com/dmd/): First-person accounts from people and families living with Duchenne muscular dystrophy.
- [FSHD Stories](https://www.dyne-tx.com/fshd/): First-person accounts from people and families living with facioscapulohumeral muscular dystrophy.
- [Resources](https://www.dyne-tx.com/resources/): Curated list of U.S. and international patient advocacy organizations and information sources for DM1, DMD and FSHD.

## Patient stories

- [Meet BillyDean and Suzette (DM1)](https://www.dyne-tx.com/dm1-story-billydean/): A mother's advocacy and her son's path to a DM1 diagnosis.
- [Meet Joachim (DM1)](https://www.dyne-tx.com/dm1-story-joachim/): One person's journey with myotonic dystrophy type 1.
- [Meet Loraine (DM1)](https://www.dyne-tx.com/dm1-story-loraine/): A family's multigenerational experience of DM1.
- [Meet Ravi (DMD)](https://www.dyne-tx.com/dmd-story-ravi/): A law student's journey with Duchenne muscular dystrophy.
- [Meet Laura and Chelsea (FSHD)](https://www.dyne-tx.com/fshd-story-chelsea/): A mother and daughter's joint perspective on living with FSHD.

## Letters to the community

- [Dyne Receives FDA Clearance to Start Early-Stage FSHD Clinical Trial](https://www.dyne-tx.com/early-stage-fshd-clinical-trial/): July 28, 2026. FDA clearance of the IND for DYNE-302 and the design of the planned Phase 1 trial.
- [Together in Progress: FDA Accepts BLA for Z-Rostudirsen and Grants Priority Review](https://www.dyne-tx.com/fda-accepts-bla-for-z-rostudirsen/): July 20, 2026. BLA acceptance, Priority Review, PDUFA target action date of January 21, 2027.
- [Zeleciment Rostudirsen Application Submitted for U.S. Accelerated Approval; Phase 3 Confirmatory FORZETTO Trial Initiated](https://www.dyne-tx.com/forzetto-trial-initiated/): May 26, 2026. BLA submission and the design of the FORZETTO Phase 3 trial.
- [New Clinical Trial Opportunity in DM1: HARMONIA Phase 3 Study Evaluating Zeleciment Basivarsen](https://www.dyne-tx.com/new-clinical-trial-opportunity-in-dm1/): April 16, 2026. HARMONIA trial design and enrolment information for the DM1 community.
- [A Historic Step Forward: Duchenne Added to Newborn Screening](https://www.dyne-tx.com/community-letter-duchenne-added-to-newborn-screening/): December 16, 2025. Duchenne muscular dystrophy added to the U.S. Recommended Uniform Screening Panel.
- [Positive Topline Results from the Phase 1/2 DELIVER Trial of Z-Rostudirsen in DMD](https://www.dyne-tx.com/community-letter-deliver-clinical-trial/): December 8, 2025. DELIVER topline results, 24-month durability data and long-term safety.
- [To the Duchenne Muscular Dystrophy Community](https://www.dyne-tx.com/community-letter-duchenne-muscular-dystrophy-community/): August 4, 2025. FDA grants Breakthrough Therapy Designation to DYNE-251.

## For healthcare professionals

- [Dyne Medical Central](https://dynemedicalcentral.com/): Dyne's dedicated resource for U.S. healthcare professionals.

## Optional

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- [Facebook](https://www.facebook.com/DyneTherapeutic/)
- [YouTube](https://www.youtube.com/@dynetherapeutics)