Entrada Therapeutics Receives Authorization in the European Union to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-45 in Patients Living with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping

Core Insights - Entrada Therapeutics is set to initiate the ELEVATE-45-201 clinical study in Q3 2025, following authorization from health authorities in multiple countries under the EU Clinical Trial Regulation [1][2] - ELEVATE-45-201 is a Phase 1/2 study focusing on ENTR-601-45, targeting patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 45 skipping, addressing a significant unmet medical need [2][4] Company Overview - Entrada Therapeutics is a clinical-stage biopharmaceutical company developing a new class of medicines aimed at intracellular targets, utilizing its proprietary Endosomal Escape Vehicle (EEV™) technology [5] - The company is advancing a robust pipeline of RNA- and protein-based programs for neuromuscular and ocular diseases, with a focus on DMD therapies targeting exon 44, 45, 50, and 51 skipping [5] Clinical Study Details - ELEVATE-45-201 is a global, two-part, randomized, double-blind placebo-controlled study evaluating the safety, tolerability, and effectiveness of ENTR-601-45 in ambulatory DMD patients [2] - Part A of the study will involve approximately 24 patients, with dosing every six weeks across three cohorts, ranging from 5 mg/kg to 15 mg/kg [2] - Part B will further assess the optimal dose for safety and efficacy, including functional outcomes and quality of life measures [2] Product Information - ENTR-601-45 is a proprietary EEV™-conjugated phosphorodiamidate morpholino oligomer (PMO) designed to address the underlying cause of DMD by restoring the mRNA reading frame for dystrophin protein production [3] - The therapy targets a specific subpopulation of DMD patients who are amenable to exon 45 skipping, with the potential to produce a functional but slightly shortened dystrophin protein [3] Disease Context - Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by mutations in the DMD gene, leading to inadequate dystrophin production and progressive muscle weakness [4] - An estimated 41,000 individuals in the U.S. and Europe are affected by DMD, with approximately 9% being amenable to exon 45 skipping [4]