Dyne Therapeutics

What Dyne Therapeutics does

Dyne Therapeutics is a clinical-stage biotechnology company developing nucleic acid therapies for genetically driven rare neuromuscular diseases. The company's core technology is the proprietary FORCE™ (Fab conjugated Oligonucleotide Repeatable Engine) platform, which conjugates therapeutic oligonucleotides to TfR1-binding antibody fragments to achieve targeted delivery to muscle tissue, addressing a long-standing barrier to muscle-directed nucleic acid therapeutics. Preclinical data also indicate the platform can cross the blood-brain barrier with approximately 75% MAPT RNA knockdown in nonhuman primates, signaling potential extension into CNS indications.

Dyne's pipeline includes two clinical-stage assets — zeleciment rostudirsen (DYNE-251) for Duchenne muscular dystrophy patients amenable to exon 51 skipping, which met its primary endpoint with 5.46% dystrophin expression and demonstrated functional improvement across all six endpoints at 24 months in the DELIVER trial; and zeleciment basivarsen (DYNE-101) for myotonic dystrophy type 1, now in the Phase 3 HARMONIA trial. Preclinical programs DYNE-302 (FSHD) and DYNE-401 (Pompe disease) extend the FORCE platform into additional indications. The company submitted a BLA for z-rostudirsen to the FDA in May 2026 with a target Q1 2027 U.S. launch.

Dyne is pre-commercial and derives no material product revenue. The company intends to commercialize via specialty pharmacy and 3PL distribution, field-based sales and medical affairs teams, and a patient services hub supporting HCP-administered infusion in rare disease centers. Pricing has not been disclosed, but the addressable populations are narrow (~1,600 U.S. patients for DMD exon 51, ~40,000 U.S. and ~55,000 EU patients for DM1). The business is funded by approximately $1.1 billion in cash at year-end 2025, supplemented by equity offerings and a Hercules Capital term loan facility of up to $400 million, providing runway into Q1 2028 ahead of first potential product revenue.

Name

Dyne Therapeutics

Legal name

Dyne Therapeutics, Inc.

Website

https://dyne-tx.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

251–500 employees

Short description

Dyne Therapeutics is a clinical-stage biotechnology company developing nucleic acid therapies for rare neuromuscular diseases using its proprietary FORCE™ muscle-targeted delivery platform, with lead programs for Duchenne muscular dystrophy and myotonic dystrophy type 1 targeting commercial launch in 2027.

Ownership category

akta.pro rank

Product category

Neuromuscular Disease Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE)

akta.pro secondary industries

Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

Keywords

• Nucleic acid therapeutics
• Neuromuscular disease therapies
• Antisense oligonucleotide drugs
• Rare disease biopharmaceuticals
• Targeted muscle delivery

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel

Revenue model

1. Pharmaceutical Product Sales: Pre-commercial biotech company expecting to generate revenue from product sales following FDA approval. Lead products z-rostudirsen (DMD) and z-basivarsen (DM1) planned for potential launch in Q1 2027 and early 2028 respectively.
2. No Current Revenue: Clinical-stage biotech with no commercial revenue as of FY2025. Reported net loss of $446.2 million with less than $1 million in revenue for FY2025.

Pricing tiers

Go-to-market motion2 records

Distribution channels3 records

Marketing channels5 records

Core offering

Dyne Therapeutics is a clinical-stage biotechnology company developing nucleic acid therapeutics for serious, genetically driven neuromuscular diseases using its proprietary FORCE platform. Its pipeline targets Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Pompe disease via antisense oligonucleotides conjugated to TfR1-binding antibody fragments for muscle-targeted delivery.

Product overview

Dyne Therapeutics is a clinical-stage biotechnology company developing nucleic acid therapies for rare genetically driven neuromuscular diseases using its proprietary FORCE platform. The company's portfolio includes two clinical-stage programs: zeleciment rostudirsen (DYNE-251) for Duchenne muscular dystrophy (DMD) and zeleciment basivarsen (DYNE-101) for myotonic dystrophy type 1 (DM1). The FORCE platform enables targeted delivery of oligonucleotides to muscle tissue, overcoming previous delivery limitations. The company also has preclinical programs including DYNE-302 for facioscapulohumeral muscular dystrophy (FSHD) and DYNE-401 for Pompe disease.

Differentiator

Problem solved

Functional benefit

Products and services

• Zeleciment rostudirsen (DYNE-251) Antisense oligonucleotide therapy designed for patients with Duchenne muscular dystrophy amenable to exon 51 skipping. Delivered via the FORCE platform using TfR1-binding antibody fragments conjugated to therapeutic oligonucleotides. Has demonstrated 5.46% dystrophin expression at 6 months and functional improvement across all six endpoints in the DELIVER trial, with cardiopulmonary benefits through 24 months.
• Zeleciment basivarsen (DYNE-101) Therapeutic candidate for myotonic dystrophy type 1 (DM1), a rare progressive neuromuscular disease. Leverages the FORCE platform for targeted muscle delivery. Has demonstrated early and sustained improvements in myotonia, muscle strength and function with favorable safety profile. Holds FDA Breakthrough Therapy, Orphan Drug, and Fast Track designations.
• DYNE-302 Preclinical therapeutic program for facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle disease affecting face, shoulders, and upper arms with no approved disease-modifying treatments. Developed using the FORCE platform for muscle-targeted delivery.
• DYNE-401 Preclinical therapeutic program for Pompe disease, a rare lysosomal storage disorder affecting muscle tissue. Developed using the FORCE platform to address progressive muscle weakness due to glycogen accumulation and respiratory failure.

Quantifiable outcome

• 5.46% dystrophin expression at 6 months in DELIVER trial (primary endpoint met)
• +3 more outcomes

Named customers2 records

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

One partnership is on record.

• Myotonic Dystrophy FoundationstrategicStrategic or Co-development Partner · 1 April 2026Collaboration on DM1 health insurance literacy research and patient advocacy initiatives. Foundation contributed to study design and interpretation of results. Joint poster presentation at AMCP 2026 Annual Conference.

Scale indicators8 records

Recent moves6 records

Expansion highlights5 records

Emerging players

• Edgewise Therapeutics: Clinical-stage biotech developing therapeutics for muscular dystrophies including DMD and Becker, sharing Dyne's muscle-disease focus and rare neuromuscular patient base.
• Solid Biosciences: Gene therapy company pursuing DMD with alternative modality to Dyne's ASO approach but same exon-skipping/genetic correction strategy and patient population.
• PepGen: Clinical-stage company developing conjugated oligonucleotide therapeutics for DMD and DM1, directly overlapping Dyne's lead indications and delivery-conjugate approach.
• Capricor Therapeutics: Cell therapy developer for DMD and other rare diseases, targeting the same patient and prescriber base as Dyne with a distinct but adjacent modality.

Direct peers

• PTC Therapeutics: Commercial-stage rare disease company with Translarna (ataluren) and other DMD/rare neuromuscular assets targeting the same patient and prescriber base as Dyne.
• Ionis Pharmaceuticals: Antisense oligonucleotide platform company and the originator of ASO chemistry used in many muscle/neuromuscular programs. Highly comparable core technology to Dyne's FORCE-conjugated ASO approach.
• Sarepta Therapeutics: The leading commercial-stage DMD therapeutic company, with approved exon-skipping ASOs (Exondys 51, Vyondys 53, Amondys 45) and gene therapy Elevidys. Directly comparable to Dyne given overlap in DMD exon-skipping modality and patient population.

Broad incumbents

• Alnylam Pharmaceuticals: Established RNAi therapeutics platform company with approved rare disease drugs and ongoing neuromuscular/hATTR programs. Comparable nucleic-acid delivery franchise and rare-disease commercial model.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with approved neuromuscular and metabolic assets, sharing Dyne's commercial-build playbook for specialty-distributed orphan therapeutics.
• BioMarin Pharmaceutical: Established rare disease commercial-stage company with neuromuscular and gene therapy assets, providing comparable scale of rare-disease launch infrastructure and orphan commercialization expertise.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/dyne_tx
• YouTubeyoutube.com/@dynetherapeutics
• Facebookfacebook.com/DyneTherapeutic
• LinkedInlinkedin.com/company/dynetx

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds11 records

Investors10 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Dyne Therapeutics