Dyne Therapeutics, Inc. · NASDAQ:DYN
Dyne Therapeutics — 44th Annual J.P. Morgan Healthcare Conference 2026
31 slides from Dyne Therapeutics's conference presentation, Jan 12, 2026
Slide 1: Advancing Clinically Validated Platform Toward Commercialization
Advancing Clinically Validated Platform Toward Commercialization 44TH ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE | JANUARY 2026
Slide 2: Forward-Looking Statements & Disclaimer
Forward-Looking Statements & Disclaimer This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this presentation, including statements regarding Dyne’s strategy, future operations,
Slide 3: Poised to Unlock Significant Commercial Opportunities in Multiple Rare Neuromuscular Diseases
Poised to Unlock Significant Commercial Opportunities in Multiple Rare Neuromuscular Diseases Positive topline results from registrational cohort in DMD Ongoing registrational cohort in DM1 NEAR-TERM VALUE DRIVERS STRONG FINANCIAL POSITION LATE-STAGE CLINICAL PIPELINE Steady cadence of expected data
Slide 4: Leveraging Our FORCE Platform for Targeted Delivery
Leveraging Our FORCE™ Platform for Targeted Delivery LINKER Connects Fab and payload PAYLOAD Modularity enables rational selection of payload to target the genetic basis of disease (ASO, PMO, siRNA, enzyme) ANTIBODY Proprietary Fab against TfR1 to enable targeted delivery to muscle and CNS Skeletal
Slide 5: FORCE Enabled Oligonucleotide Delivery to Muscle and CNS
FORCE Enabled Oligonucleotide Delivery to Muscle and CNS Notes for PMO: Results after repeat IV dose of naked ASO or DYNE-251 in male cynomolgus monkeys, 2 x 30 mg/kg on day 0 and day 7, analyzed day 28; n = 4 – 5. Notes for ASO: Results after a single IV dose of naked ASO or FORCE in male cynomolgu
Slide 6: Potential of the FORCE Platform Validated by Recent Z-Rostudirsen Topline Clinical Results
Potential of the FORCE Platform Validated by Recent Z-Rostudirsen Topline Clinical Results Validation with Z-Rostudirsen DELIVER Data 1. Data as of August 19, 2025. 2. No participants have demonstrated persistent related anemia with Hgb levels <11.2 g/dL (threshold for anemia in children (ref: Power
Slide 7: Neuromuscular Pipeline Leveraging Clinically Validated Platform
Neuromuscular Pipeline Leveraging Clinically Validated Platform Pipeline Expansion Opportunities CNS, Rare skeletal, Cardiac, Metabolic DISEASE TARGET PRECLINICAL PHASE 1/2 ESTIMATED PATIENTS Myotonic dystrophy type 1 (DM1) DMPK US: ~40,000 EU: ~55,000 Duchenne muscular dystrophy (DMD) Exon 51 US: ~
Slide 8: Broad and Durable Functional Improvement Observed with Z-Rostudirsen in DMD
Broad and Durable Functional Improvement Observed with Dyne's First Clinically Validated Program: Z-Rostudirsen in DMD
Slide 9: Potential Best-in-class Targeted Exon Skipping
Potential Best-in-class Targeted Exon Skipping Increase dystrophin expression and enable less frequent dosing to deliver functional improvement OUR APPROACH ~12,000 (US) ~16,000 (EU) ~ 13% is exon 51 skip amenable1 DMD Population Mutation in DMD gene for dystrophin Exon 51 skip amenable DMD is a par
Slide 10: DELIVER Study to Support Accelerated Approval of Z-Rostudirsen in DMD
DELIVER Study to Support Accelerated Approval of Z-Rostudirsen in DMD Selection of registrational dose (20 mg/kg Q4W) based on multiple ascending dose (MAD) data Q2 2026 Planned submission for U.S. Accelerated Approval based on positive results from Registrational Expansion Cohort Functional imp
Slide 11: Z-Rostudirsen Achieved Statistically Significant and Robust Increase in Dystrophin Expression at 6M
Z-Rostudirsen Achieved a Statistically Significant and Robust Increase in Dystrophin Expression at 6M in Registrational Expansion Cohort 1. Biopsies taken approximately 28 days after most recent dose. 2. Based on geometric mean. 3. One REC placebo participant sample could not be analyzed at Week 25.
Slide 12: Improvement in TTR Velocity and 10MWR Velocity Relative to Baseline and Placebo
Improvement in TTR Velocity and 10MWR Velocity at 6 Months Relative to Baseline and Placebo Time to Rise1 (TTR) Velocity2 10-Meter Walk/Run (10MWR) Velocity2 Placebo (REC+MAD) (n=18) Z-rostudirsen 20 mg/kg Q4W (REC) (n=21) Placebo (REC+MAD) (n=18) Z-rostudirsen 20 mg/kg Q4W (REC) (n=21) ∆0.2 m/sec a
Slide 13: Improvement in NSAA and SV95C Relative to Baseline
Improvement in NSAA and SV95C at 6 Months Relative to Baseline North Star Ambulatory Assessment (NSAA)1 Stride Velocity 95th Centile (SV95C)1 Improvement ∆2.1 points approaches MCID2 (nominal p=0.0894)3 1. Ambulant participants; missing values imputed. 2. Ayyar Gupta et al. PLoS One. 2023 Apr 26;18(
Slide 14: Improvement in PUL2.0; Preservation of Lung Function at 6 Months
Improvement in PUL2.0 at 6 Months Relative to Baseline and Placebo; Preservation of Lung Function at 6 Months Forced Vital Capacity Percent Predicted (FVC%p)1 Placebo (REC+MAD) (n=23) Z-rostudirsen 20 mg/kg Q4W (REC) (n=22) Placebo (REC+MAD) (n=20) Z-rostudirsen 20 mg/kg Q4W (REC) (n=15) 1. Ambulant
Slide 15: Unprecedented Breadth and Durability of Functional Improvement with Z-Rostudirsen
Unprecedented Breadth and Durability of Functional Improvement with Z-Rostudirsen BREADTH DURABILITY Endpoint Patient Population Muscle System 6-month Functional Improvement vs. Placebo1 24-month Functional Improvement vs. Baseline2 TTR Velocity Ambulatory Trunk & lower limbs 10MWR Velocity Ambu
Slide 16: Z-Rostudirsen: Favorable Safety Profile
Z-Rostudirsen: Favorable Safety Profile Summary of treatment-emergent adverse events (TEAEs)1 1,441 doses of z-rostudirsen administered to date representing 113 patient-years of follow-up (up to 36 months)1 1,062 doses of z-rostudirsen at 20 mg/kg dose level administered to date1 Most related TEAEs
Slide 17: Z-Rostudirsen Offers a Compelling Profile for Potential Accelerated Approval in DMD
Z-Rostudirsen Offers a Compelling Profile for Potential Accelerated Approval and Addressing Unmet Need in DMD Z-Rostudirsen: Potential Best-in-class Profile for Exon 51 DMD Statistically Significant and Robust Increase in Dystrophin at 6 Months Favorable Safety & Tolerability Profile up to 36 Months
Slide 18: Preparing to Launch into an Established Rare Disease Market
Preparing to Launch into an Established Rare Disease Market with Well-Characterized Patient Population and Treatment Centers Significant overlap expected with DM1 centers First exon skipper approved in 2016 Established reimbursement pathways and clear recognition of unmet need by payers ~ 55% Medica
Slide 19: Capital-Efficient Commercial Infrastructure
Capital-Efficient Commercial Infrastructure Designed to Deliver Potentially Transformative Therapies and Optimize Value Strong expertise in rare neurological and neuromuscular diseases Broad US launch experience including SPINRAZA, ELEVIDYS, VYONDYS, AMONDYS, and DAYBUE Deep expertise across Medical
Slide 20: De-Risked Opportunity to Build a Broader DMD Franchise
De-Risked Opportunity to Build a Broader DMD Franchise Potentially Tripling Addressable Patient Population May not be amenable to exon skipping – ~20% Exon 51 – 13% Exon 53 – 8% Exon 45 – 8% Exon 44 – 6% Exon 50 – 4% Other exon skips – ~30% Exon 52 – 4% Exon 43 – 4% Exon 55 – 2% Exon 8 – 2% Approxim
Slide 21: Drivers for Potential Shareholder Value Creation in DMD
Drivers for Potential Shareholder Value Creation in DMD Significant unmet needs despite approved therapies Identified patient population Pricing precedent with established reimbursement Concentrated treatment centers attractive market characteristics Wholly-owned assets Potential best-in-class profi
Slide 22: Second Lead Program: Z-Basivarsen in DM1
Second Lead Program Leveraging Clinically Validated Platform for Larger Adjacent Indication: Z-Basivarsen in DM1
Slide 23: Restore normal RNA splicing to achieve functional improvement for those living with DM1
Restore normal RNA splicing to achieve functional improvement for those living with DM1 Functional Improvement via Splicing Correction in Nucleus OUR APPROACH Mutation in the DMPK gene leads to mis-splicing of multiple genes Onset at any point, depending on DM1 phenotype Life expectancy of 45 - 60 y
Slide 24: ACHIEVE Study to Support Accelerated Approval of Z-Basivarsen in DM1
EarlyQ3 2027 ACHIEVE Study to Support Accelerated Approval of Z-Basivarsen in DM1 Selection of registrational dose (6.8 mg/kg Q8W) based on multiple ascending dose (MAD) data Potential submission for U.S. Accelerated Approval based on data from Registrational Expansion Cohort (REC) Proof-of-
Slide 25: Z-Basivarsen at 6.8 mg/kg Q8W Improved Foundational Pathobiology of DM1 at 3 Months
Z-Basivarsen at 6.8 mg/kg Q8W Improved Foundational Pathobiology of DM1 at 3 Months Note: One baseline sample in 6.8 mg/kg treatment groups not included within splicing assay as the sample did not meet QC criteria. 3 months = 85 days. CASI-22 = composite alternative splicing index. n = 5 DMPK % Mean
Slide 26: Robust and Sustained vHOT Improvement with Z-Basivarsen
Robust and Sustained vHOT Improvement with Z-Basivarsen at 6 and 12 Months vHOT Middle Finger Improvement 1. vHOT Middle Finger (sec) is the average of all myotonia trials for an individual participant in ACHIEVE; vHOT = video hand opening time; SEM = standard error of the mean; BL = baseline. 3 mon
Slide 27: Broad and Sustained Functional and Patient-Reported Improvement through 12 Months
Broad and Sustained Functional and Patient-Reported Improvement with Z-Basivarsen through 12 Months Quantitative Muscle Testing (QMT) Total Score Improvement 10-Meter Walk/Run Test 5 Times Sit to Stand Improvement Improvement Improvement Myotonic Dystrophy Health Index (MDHI) Total Score BL, baselin
Slide 28: Sustained Improvement in CNS-Related MDHI Subscales
Cognitive Impairment Communication Emotional Issues Sleep Pain Fatigue Improvement Subscale Change from Baseline (Mean +/- SEM) SEM = standard error of the mean; BL = baseline; Patient-reported outcomes (PRO) including Myotonic Dystrophy Health Index (MDHI) collected at baseline, 6 months (169 days)
Slide 29: Z-Basivarsen Safety: TEAE Summary
TEAE Category Participants with ≥1 TEAE – n (%) 1.8 mg/kg Q4W+Rec. N=16 3.4 mg/kg Q4W+Rec. N=16 3.4 mg/kg Q8W N=8 5.4 mg/kg Q8W N=8 6.8 mg/kg Q8W N=8 Overall (N=56) Any TEAE 16 (100%) 16 (100%) 8 (100%) 8 (100%) 8 (100%) 56 (100%) Any related TEAE 9 (56%) 10 (63%) 3 (38%) 6 (75%) 6 (75%) 34 (61%) An
Slide 30: Transforming Dyne into a Commercial Organization as Early as 2027
Transforming Dyne into a Commercial Organization as Early as 2027 Q1 2025 Completed enrollment of Registrational Expansion Cohort Early Q2 2026 Complete enrollment planned for Registrational Expansion Cohort December 2025 Positive topline results from Registrational Expansion Cohort Q1 2027 Data pla
Slide 31: Q&A
THERAPEUTICS Q&A
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