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Entrada Therapeutics reports positive Phase 1/2 data for ENTR-601-45 in Duchenne muscular dystrophy
TRDAEntrada Therapeutics, Inc.
Entrada Therapeutics announced positive topline results from Cohort 1 of the Phase 1/2 ELEVATE-45-201 study evaluating ENTR-601-45, an investigational therapy for Duchenne muscular dystrophy (DMD) patients amenable to exon 45 skipping. The cohort met primary objectives with favorable safety and tolerability at a 5 mg/kg dose. Secondary endpoints showed a mean increase of 0.10% in dystrophin production and 0.15% in exon skipping versus baseline. Plasma exposures aligned with preclinical predictions, supporting escalation to higher doses. Cohort 2 enrollment is complete, with data expected in the first half of 2027. Entrada Therapeutics operates as a clinical-stage biopharmaceutical company developing genetic medicines for neuromuscular and inherited retinal diseases.