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

Company Updates - Entrada Therapeutics is set to initiate the ELEVATE-45-201 study in Q3 2025, focusing on the treatment of Duchenne muscular dystrophy (DMD) [1][2] - The company has received regulatory authorization from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) for the ELEVATE-45 program, with ongoing regulatory review in the EU [1][2] - ELEVATE-45 is the second of three novel exon skipping programs that Entrada plans to advance into global clinical development in 2025 [1] Clinical Study Details - ELEVATE-45-201 is a Phase 1/2 multiple ascending dose (MAD) clinical study evaluating the investigational product ENTR-601-45 for DMD patients with specific mutations [2] - The study will consist of two parts: Part A will assess safety and pharmacokinetics in approximately 24 patients, with dosing every six weeks ranging from 5 mg/kg to 15 mg/kg [2] - Part B will further evaluate the optimal dose for safety and efficacy, including functional outcomes and quality of life measures [2] Product Information - ENTR-601-45 is a proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO) targeting the underlying cause of DMD due to mutations in the DMD gene [3] - The therapy aims to restore the mRNA reading frame to allow for the production of a functional, albeit shortened, dystrophin protein [3] Disease Overview - Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by mutations in the DMD gene, leading to insufficient dystrophin production, which is critical for muscle cell integrity [4] - An estimated 41,000 individuals in the U.S. and Europe are affected by DMD, resulting in progressive muscle weakness and associated complications [4] Company Vision - Entrada Therapeutics is a clinical-stage biopharmaceutical company focused on developing a new class of medicines that target previously inaccessible intracellular sites [5] - The company is advancing a diverse portfolio of RNA, antibody, and enzyme-based programs for various diseases, including neuromuscular and metabolic disorders [5]