ENTR-601-44 / Entrada - LARVOL DELTA

Endosomal escape vehicle platform safely and effectively delivers oligonucleotide therapeutics to skeletal and cardiac muscle tissue for the potential treatment of Duchenne muscular dystrophy (ACS-Fall 2025) - "Additionally, healthy human volunteers administered a single dose of ENTR-601-44, a DMD exon 44 skipping EEV-PMO construct, showed dose-dependent exon 44 skipping with no adverse events related to study drug. These findings demonstrate the therapeutic potential of the EEV platform and suggest further clinical development of EEV-PMO constructs in people living with DMD."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

ELEVATE-44: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: Entrada Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

ELEVATE-44: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: Entrada Therapeutics, Inc.

New P1/2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Clinical Trial of ENTR-601-44, an Endosomal Escape Vehicle (EEV)-conjugated PMO, for the Treatment of Duchenne Muscular Dystrophy (ASGCT 2025) - "Supported by the Musculoskeletal Cell and Gene Therapy Committee"

Clinical • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy • Musculoskeletal Diseases

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 (GlobeNewswire) - "Entrada Therapeutics...announced it has received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee to initiate ELEVATE-45-201. ELEVATE-45-201 is a Phase 1/2 multiple ascending dose (MAD) clinical study to evaluate Entrada’s investigational medicinal product ENTR-601-45 for the potential treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 45 skipping...'Building on the momentum of our ELEVATE-44 program, this authorization brings us closer to having three Duchenne programs in clinical development by the end of this year.'...Study participants may be eligible to enter an open label extension study (OLE), in which the safety, efficacy and tolerability of ENTR-601-45 will be evaluated over a longer period of time. The Company is on track to initiate ELEVATE-45-201 in Q3 2025."

New P1/2 trial • Pipeline update • Duchenne Muscular Dystrophy

Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44 (GlobeNewswire) - "Entrada Therapeutics, Inc...today announced that the United States Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping...The Company plans to initiate study enrollment in the first half of 2026, which would enable a seamless transition into an OLE."

FDA event • New P1 trial • Duchenne Muscular Dystrophy

Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-44-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of ENTR-601-44 in Patients Living with Duchenne Muscular Dystrophy (GlobeNewswire) - "Entrada Therapeutics, Inc...today announced it had received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee for its Clinical Trial of an Investigational Medicinal Product to initiate ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44 for the potential treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping...The Company is on track to initiate ELEVATE-44-201 in Q2 2025."

New P1/2 trial • Duchenne Muscular Dystrophy