Dyne Therapeutics (NASDAQ:DYN) executives said the company is shifting from data generation to regulatory execution and commercial preparation as it advances programs in Duchenne muscular dystrophy, myotonic dystrophy type 1 and facioscapulohumeral muscular dystrophy.
Speaking at RBC Capital Markets’ 2026 Global Healthcare Conference, Chief Financial Officer Erick J. Lucera said Dyne views 2025 as “a year of data de-risking” and 2026 as “a year of execution and preparation for commercial launches.” He said the company is preparing for a potential DMD launch, followed by DM1, while continuing to build a pipeline based on its FORCE platform.
Dyne Highlights DMD Regulatory Path and Confirmatory Trial
Lucera said Dyne recently held a pre-BLA meeting with the FDA for its DMD program and remains on track to submit a biologics license application this quarter. He said the company has been aligned with the agency on dystrophin as an endpoint and has assumed Priority Review in its timelines.
Chief Scientific Officer Ranjan “Ron” Batra said the FDA’s accelerated approval pathway remains open to Dyne based on dystrophin as a surrogate endpoint, despite recent confirmatory trial setbacks for other approved exon-skipping therapies in the DMD field. Batra said Dyne’s DELIVER study showed statistically significant dystrophin data, citing a p-value of 0.0001%, along with functional improvement in endpoints such as rise from floor velocity.
Batra also discussed Dyne’s confirmatory DMD trial, which he said has been initiated. The study is designed to enroll 90 patients randomized one-to-one, with rise from floor velocity as the primary endpoint at 72 weeks. He said the trial is “fully powered” and may also support ex-U.S. applications.
Batra said Dyne selected rise from floor velocity because difficulty getting up from the floor is clinically meaningful in DMD and because the company’s prior data support the endpoint. He also noted that the broader DMD field is moving toward similar functional measures.
Commercial Planning and Additional Exons
On the commercial side, Lucera said DMD is an attractive launch market because pricing and reimbursement infrastructure already exist, patients are highly motivated and advocacy organizations are strong. He declined to discuss specific pricing plans, saying it is too early, but said Dyne believes its once-monthly dosing schedule could offer an advantage over once-weekly alternatives.
Lucera also discussed Dyne’s plans beyond exon 51, including work on other exons such as 53, 45 and 44. He said the programs use the same Fab, linker and oligonucleotide chemistry, and would involve many of the same patients, centers and physicians. He said bringing four additional exons into the clinic could triple Dyne’s total addressable market, though the company has not yet detailed a regulatory strategy.
DM1 Program Focuses on vHOT and HARMONIA
Dyne also addressed its DM1 program, including a trial that could support accelerated approval using vHOT as an intermediate clinical endpoint. Lucera said the company had always believed 60 patients would be sufficient for powering, but the final enrollment could exceed that number because of patient demand and the company’s ethical obligation to continue patients already in the screening funnel. He said the increase is not tied to operational or statistical needs.
Batra cautioned against direct comparisons between Dyne’s DM1 candidate, zeleciment basivarsen, and Novartis’ program, citing differences in mechanism of action. He said Dyne uses an antisense oligonucleotide intended to target mutant DMPK in the nucleus, while Novartis uses an siRNA approach. He also said Dyne’s Fab structure may provide better tissue penetration than a monoclonal antibody and that Dyne has not seen anemia in the clinic.
For Dyne’s HARMONIA confirmatory trial in DM1, Batra said the company selected five-times sit-to-stand as the primary endpoint because it captures multiple clinically relevant aspects of the disease, including leg strength, myotonia, trunk and core strength, posture and fall risk. Lucera said the company has not yet provided timing for HARMONIA data.
FSHD Program Remains Early
Batra said Dyne’s FSHD program is expected to be the next to enter the clinic, though the company has not provided timing guidance. He said the program could benefit from the FORCE platform’s Fab-based tissue penetration and potential access to satellite cells, which he described as important in FSHD.
Lucera said FSHD represents a significant unmet need and could leverage Dyne’s manufacturing and future commercial infrastructure, given overlap with centers treating DM1 and DMD patients. He said the company remains excited about the program but has not disclosed go/no-go timing or a biomarker strategy.
Asked what investors may be underappreciating about Dyne, Lucera said the company’s story is centered on execution: bringing DMD and DM1 programs toward market and moving additional molecules into the clinic.
About Dyne Therapeutics (NASDAQ:DYN)
Dyne Therapeutics is a clinical-stage biotechnology company specializing in the development of localized gene regulation therapies for serious rare diseases. The company’s proprietary FORCE (Facilitated Orthogonal Receptor?mediated Cargo Evaluation) platform is designed to enable targeted delivery of oligonucleotide and gene therapy modalities to skeletal and respiratory muscles. Dyne’s lead programs focus on Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD), with preclinical and early clinical studies evaluating safety, tolerability and tissue specificity.
Since its founding in 2019 by Flagship Pioneering, Dyne has advanced multiple product candidates using its modular delivery approach, which couples engineered ligands with therapeutic payloads to improve uptake into muscle cells.
