Dyne Therapeutics
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Dyne Therapeutics (DYN) investor relations material

Dyne Therapeutics 44th Annual J.P. Morgan Healthcare Conference summary

Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
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44th Annual J.P. Morgan Healthcare Conference summary15 Jan, 2026

Strategic vision and platform advancement

  • Advancing a clinically validated platform for rare neuromuscular diseases, with two late-stage assets in DMD and DM1 and near-term value drivers including BLA submission and product launches in 2026–2027.

  • FORCE platform enables targeted delivery to muscle and CNS using proprietary Fab against TfR1, supporting multiple payloads and broad pipeline expansion.

  • Human and non-human primate data show superior delivery and functional improvement, especially with DYNE-251 in DMD.

  • Cash position of ~$1.1 billion as of 12/31/25, with runway into Q1 2028 and all assets fully owned.

  • Platform enables capital-efficient expansion and de-risked pipeline, aiming to triple DMD market size.

Clinical pipeline and data highlights

  • DELIVER trial for DYNE-251 in DMD showed a seven-fold increase in dystrophin and significant functional improvements at six months, with robust results across TTR velocity, 10MWR, NSAA, SV95C, PUL2.0, and FVC %p.

  • Durable improvements maintained up to 36 months; safety profile favorable with most adverse events mild or moderate and over 1,000 doses administered.

  • DYNE-101 in DM1 demonstrated 33% DMPK knockdown, 25% splicing correction, and functional improvements in vHOT, sit-to-stand, 10-meter walk, and patient-reported outcomes through 12 months.

  • Quantitative muscle testing showed 10–20% strength increase; MDHI subscales indicated CNS impact at higher doses.

  • No serious related adverse events for DYNE-101; most TEAEs were mild or moderate, with 93 patient years and over 1,000 doses.

Regulatory and commercialization plans

  • Planned U.S. Accelerated Approval submission for DYNE-251 in DMD in Q2 2026, with potential commercial launch in Q1 2027.

  • DYNE-101 in DM1 targeting Accelerated Approval submission in early Q3 2027, with Phase 3 studies planned for global approval.

  • Breakthrough designation for both DMD and DM1, with ongoing FDA dialogue and alignment on accelerated approval pathways.

  • Commercial infrastructure, management, and supply chain in place for 2027 launch; gross margins expected to be competitive with rare disease antibody companies.

  • Well-characterized patient populations and concentrated treatment centers support efficient market entry and reimbursement.

How will Z-Rostudirsen redefine DMD treatment?
What specific CNS endpoints for DM1 Phase III?
How does platform validation accelerate pipeline?
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Q4 202527 Feb, 2026
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Frequently asked questions

Dyne Therapeutics Inc. is a biotechnology firm engaged in the development of innovative therapeutics aimed at addressing genetically driven muscle diseases. Established in 2017 and based in Waltham, Massachusetts, the company primarily focuses on advancing a portfolio of muscle disease therapeutics. These efforts include targeting conditions such as myotonic dystrophy type 1, duchenne muscular dystrophy, facioscapulohumeral dystrophy, and other rare skeletal, cardiac, and metabolic muscle diseases. Dyne Therapeutics utilizes its proprietary FORCE platform, which is designed to enhance the delivery of disease-modifying therapeutics directly to muscle tissue. Its shares are listed on the Nasdaq.

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