Business Description

Overview We are a clinical-stage biopharmaceutical company aiming to transform the lives of patients by e stablishing a new class of medicines that engage intracellular targets that have long been considered inaccessible . Through proprietary, versatile and modular approaches, we are advancing a robust development portfolio of genetic medicines for the potential treatment of neuromuscular and inherited retinal diseases, among others. In 2026, we expect to progress our ENTR-601-44 and ENTR-601-45 clinical trials, an EU filing for the ENTR-601-50 clinical trial, and regulatory submissions for ENTR-601-51. In addition, our VX-670 partnership with Vertex Pharmaceuticals Incorporated (“Vertex”) continues to progress, with dosing completion anticipated in mid-2026. We anticipate reporting on the results of two cohorts of patient data from our ENTR-601-44 program, and one from our ENTR-601-45 program during 2026. Clinical-Stage Development Pipeline: Entrada continues to advance multiple clinical programs in people living with Duchenne muscular dystrophy (“DMD”) in the United Kingdom (“UK”), European Union (“EU”) and United States (“U.S.”). In 2026, we expect to have four clinical-stage programs in its DMD franchise (ENTR-601-44, ENTR-601-45, ENTR-601-50 and ENTR-601-51). When combined, we estimate that there are over 11,500 patients in the U.S. and Europe that carry mutations amenable to Entrada's current exon skipping programs. Complementing the ongoing clinical progress of the DMD franchise is the myotonic dystrophy type 1 (“DM1”) partnership with Vertex (“VX-670”). Each of these programs utilize the same endosomal escape vehicle, and as such, we anticipate initial data readouts from any one of the candidate clinical trials to provide critical insights for the rest. ELEVATE-44-201: The Company completed dosing of Cohort 1 of the global Phase 1/2 multiple ascending dose (“MAD”) portion of the clinical study of ENTR-601-44 in ambulatory patients living with DMD who are amenable to exon 44 skipping, and those patients transitioned to the 6 dose open label portion of the trial. Dosing every 6 weeks of all 8 patients is ongoing in this Phase 2 portion of the study. In February 2026, an independent Data Monitoring Committee (DMC) reviewed the data to date from the eight patients enrolled in Cohort 1 and recommended initiation of Cohort 2 at the increased dose of 12 mg/kg without protocol modification. The Company is on track to report data from Cohort 1 (6 mg/kg) in the second quarter of 2026, data from Cohort 2 (12 mg/kg) by year-end, and data from Cohort 3 (up to 18 mg/kg) to follow if needed. We expect to see double digit dystrophin production in the first patient cohort and, based on a combination of non-clinical and healthy normal volunteer data, a multi-fold increase in dystrophin as we dose up in the second and third cohorts. We intend to open an expansion cohort later in the year to increase the number of participants treated in the ELEVATE-44-201 study, as this study has been designed to support an accelerated approval in the U.S. Additionally, in December 2025, the U.S. Food and Drug Administration (“FDA”) granted Rare Pediatric Disease Designation to ENTR-601-44. ELEVATE-44-102: The Company believes this clinical trial, in the underserved adult patient population, would be best to initiate at the highest advisable starting dose in patients with advanced disease. Following a review of safety, PK and PD data from the ELEVATE-44-201 study in Q2 2026, we may have an opportunity to re-engage with the FDA to discuss increasing the planned doses in this clinical trial. As such, the Company will provide an update on clinical study design and timing following interactions with the FDA. ELEVATE-45-201: The Company initiated patient dosing in the global Phase 1/2 MAD portion of the clinical study of ENTR-601-45 in ambulatory patients living with DMD who are amenable to exon 45 skipping. The Company is on track to report data from Cohort 1 (5 mg/kg) in mid-2026, with data from Cohort 2 and Cohort 3 (up to 10 mg/kg and 15 mg/kg) to follow. The proposed ELEVATE-45-201 clinical trial design and dosing regimen is similar to ELEVATE-44-201, incorporating a MAD Phase 1 portion, a 6 dose open label Phase 2 portion and an expansion cohort.…