ENTR-601-45 / 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

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-45: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (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-45: A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: Entrada Therapeutics, Inc.

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

Exon 45 Skipping, Dystrophin Production, and Functional Improvement with ENTR-601-45 in Preclinical Models of Duchenne Muscular Dystrophy (ASGCT 2025) - "To assess the therapeutic potential of ENTR-601-45 in vivo, we utilized del44hDMD.mdx mice containing a human dystrophin-expressing transgene (hDMD) with DMD exon 44 deleted on an mdx background. Together, these results show the therapeutic potential of ENTR-601-45 and support further study in DMD patients amenable to exon 45 skipping. Disease Focus of Abstract:Neuromuscular Disorders"

Preclinical • CNS Disorders • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

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 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