ENTR-601-51 / Entrada - LARVOL DELTA

Preclinical Efficacy of ENTR-601-51 for the Treatment of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy (ICNMD 2026) - "Preclinical studies have established the therapeutic potential of ENTR-601-44, ENTR-601-45, and ENTR-601-50 for patients with exon 44, 45, and 50 skip-amenable DMD, respectively...mdx gastrocnemius muscle were also measured. In patient-derived skeletal muscle cell lines with DMD exon 51 skip-amenable mutations, treatment with ENTR-601-51 resulted in robust, dose-dependent exon 51 skipping and dystrophin protein expression... These results demonstrate that ENTR-601-51 is efficiently delivered to skeletal and cardiac muscle in vivo, thereby producing durable exon skipping and functional dystrophin protein that are able to rescue muscle contractile function in a relevant mouse model of human DMD . These findings support further evaluation of ENTR-601-51 in patients with DMD amenable to exon 51 skipping."

Preclinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • HSP90AA1

Entrada Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "Entrada submitted regulatory filings to initiate a global Phase 1/2 MAD clinical study of ENTR-601-45 in patients living with DMD who are amenable to exon 45 skipping in the U.K. and the EU. The Company remains on track to submit global regulatory applications for ENTR-601-50 in H2 2025 and for ENTR-601-51 in 2026....Research & Development (R&D) Expenses: R&D expenses were $33.4 million for the fourth quarter of 2024 and $125.3 million for the full year of 2024, compared to $28.3 million and $99.9 million for the same periods in 2023, respectively. The increases were primarily driven by additional costs incurred for ENTR-601-44, ENTR-601-45, and ENTR-601-50."

Commercial • New P1/2 trial • New trial • Duchenne Muscular Dystrophy