ENTR-601-44 / Entrada - LARVOL DELTA

ENTR-601-44-201: First-in-Patient Results From a Phase 1/2 Study Evaluating ENTR-601-44 in Exon 44 Skip-Amenable DMD (ICNMD 2026) - "In nonhuman primates, 4 doses of ENTR-601-44 produced 29% DMD exon 44 skipping, indicating target engagement. Topline data, including assessments of safety and tolerability, change in dystrophin and exon 44 skipping levels, and pharmacokinetics of ENTR-601-44 will be presented."

Clinical • First-in-human • P1/2 data • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

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

Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE) (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: Entrada Therapeutics, Inc.

New P2 trial • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

ENTR-601-DMD-202: A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping tonEvaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Productsn(ELEVATE-LTE) (clinicaltrialsregister.eu) - P1/2 | N=36 | Not yet recruiting | Sponsor: Entrada Therapeutics Inc.

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

ENTR-601-DMD-202: A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping tonEvaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Productsn(ELEVATE-LTE) (clinicaltrialsregister.eu) - P1/2 | N=11 | Not yet recruiting | Sponsor: Entrada Therapeutics Inc.

New P1/2 trial • 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.

Trial initiation date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

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