Dyne Therapeutics announced positive clinical data for its therapies DYNE-101 and DYNE-251 during an investor event on January 10, 2025. DYNE-101 for myotonic dystrophy type 1 (DM1) demonstrated significant splicing correction and functional improvements, leading Dyne to plan a global Registrational Expansion Cohort of the ACHIEVE trial with a registrational dose of 6.8 mg/kg Q8W and a potential U.S. Accelerated Approval submission in H1 2026.
Meanwhile, DYNE-251, targeting Duchenne muscular dystrophy (DMD), is also pursuing U.S. Accelerated Approval based on dystrophin as a surrogate endpoint, with data expected by late 2025 to support an early 2026 submission. Both programs show promise in transforming treatment paradigms for these genetic diseases.
了解更多： 杜氏肌营养不良症的治疗方法（所有研究列表）
