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2025 Muscle Wasting 1 Mahasweta Girgenrath, PhD Vice President, Neuromuscular Therapeutics 2025 International Conference on Muscle Wasting EEV-mediated Delivery to Satellite Cells: Towards a Comprehensive Correction of Pathophysiology in a Preclinical Model of Duchenne Muscular Dystrophy
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2025 Muscle Wasting 2 DISCLAIMER DMD, Duchenne muscular dystrophy; EEV, endosomal escape vehicle. This presentation has been prepared by Entrada Therapeutics, Inc. (the “Company”) and shall not constitute an offer to sell or a solicitation of an offer to buy securities or an invitation or inducement to engage in investment activity nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification of such securities under the securities law of any such jurisdiction. The Company has filed a shelf registration statement (including a prospectus) with the Securities and Exchange Commission (the “SEC”) for the offering to which this presentation relates. Before you invest in any securities of the Company, you should read the prospectus in that registration statement and any other documents the Company has filed with the SEC for more complete information about the Company and the offering. You may get these documents for free by visiting EDGAR on the SEC website at www.sec.gov. This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding the Company's strategy, future operations, prospects and plans, objectives of management, the validation and differentiation of Entrada’s approach and EEV platform and its ability to provide a potential treatment for patients, expectations regarding significant accumulation of exon skipping and dystrophin production in patients, expectations regarding the importance of endosomal escape to therapeutic index optimization, the translatability of the data from the Phase 1 clinical study for ENTR-601-44 to our planned DMD clinical studies, expectations regarding the ability of the Company’s preclinical studies and clinical studies to demonstrate safety and efficacy of its therapeutic candidates, and other positive results, expectations regarding the Company’s planned Phase 1/2 multiple ascending dose (“MAD”) clinical studies of ENTR-601-44 and -45, including their study initiations in Q2 2025 and Q3 2025, respectively, expectations regarding the Company’s planned global Phase 1/2 MAD clinical study of ENTR-601-50, including its initiation in Q4 2025, the ability to recruit for, enroll, and complete a global Phase 1/2 study for ENTR-601-44, -45, -50, and -51, the ability to recruit for, enroll, and complete a Phase 1b study for ENTR-601-44 in the US, expectations regarding the approvals and specific protocols for the Company’s planned Phase 1/2 clinical studies for ENTR-601-44, -45, and -50, the timing of regulatory filings for the planned Phase 1/2 clinical studies for ENTR-601-50 in the second half of 2025 and ENTR-601-51 in 2026, candidate selection for ENTR-601-51 in December 2024, the potential of its EEV product candidates and EEV platform, including the potential for ENTR-601-44, -45, -50, and -51 to be transformative treatment options, the continued development and advancement of ENTR-601-44, -45, -50, and -51 for the treatment of Duchenne and the partnered product VX-670 for the treatment of myotonic dystrophy type 1, and the sufficiency of the Company’s cash resources extending into 2027, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “objective,” “ongoing,” “plan,” “predict,” “project,” “potential,” “should,” or “would,” or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities and the initiation and completion of preclinical studies and clinical studies; uncertainties as to the availability and timing of results from preclinical and clinical studies; timing of and expectations regarding the Company’s ability to submit and obtain regulatory authorization and initiate clinical studies; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical studies; whether earlier clinical data will be predictive of later clinical data; our ability to establish and maintain collaborations or strategic relationships; whether the Company's cash resources will be sufficient to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in the Company's filings with the SEC, including the Company's most recent Form 10-K and in subsequent filings the Company may make with the SEC. In addition, the forward-looking statements included in this presentation represent the Company's views as of the date of this presentation. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company's views as of any date subsequent to the date of this presentation.
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2025 Muscle Wasting 3 OUR MISSION: To Treat Devastating Diseases With Intracellular Therapeutics 3 We’re proud to share the stories of JJ, Andrew, Max and Franklin – all living with Duchenne muscular dystrophy
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2025 Muscle Wasting 4 EEV TM PLATFORM
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2025 Muscle Wasting 5 ENDOSOMAL ESCAPE VEHICLE (EEV ) CONSTRUCT THERAPIES Qian, Z. et al. ACS Chem. Biol. 2013; Qian, Z. et al. Biochemistry 2014; Qian, Z. et al. Biochemistry 2016; Sahni, A. et al. ACS Chem. Biol. 2020; Pei, D. Acc. Chem. Res. 2022. High Intracellular Uptake ~90% Uptake Efficient Endosomal Escape ~50% Escape vs. ~2% Standard Unique Budding Mechanism Conserves Endosomal Integrity Unique chemistry Improved uptake and endosomal escape Cyclic structure Extended half-life and increased stability Phospholipid binding Broad biodistribution to all cells Consistent and predictable pharmacokinetics Same EEV construct used across initial programs
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2025 Muscle Wasting 6 EEV construct Therapeutic modality EEV LIBRARY: SCREENING AND OPTIMIZATION Discovery Engine for Intracellular Therapeutics • Cyclic peptide library design and combinatorial synthesis to generate EEV library • Delivery and counter-screening assays enabled for in vitro high throughput screening • Functional screening of lead EEV constructs in vivo to select for pharmacodynamic activity in target tissues • Optimize conjugation chemistry for desired therapeutic modality Screening Cascade for EEV Candidates Chemically-diverse >500 member EEV library In vitro functional validation in relevant cell types with therapeutic payload Assess in vivo functional delivery in wild-type and disease models Identify EEV candidate with desired therapeutic profile EEV constructs with robust target cell uptake and efficacy Well-tolerated EEV constructs with desired tissue functional delivery Fit-for-purpose EEV candidate for target indication EEV, endosomal escape vehicle.
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2025 Muscle Wasting 7 • EGFP654 mice were evaluated for splice correction 7 days following three weekly 10 mg/kg IV injections of PMO654 or EEV3-PMO654 IMPROVED EEV -CONSTRUCTS FOR MUSCLE DELIVERY Rational substitution of cationic residues with a surrogate results in robust functional delivery to skeletal and cardiac muscle EEV3-PMO654 Structure and Medicinal Chemistry Enhanced Functional Delivery to Muscle **p<0.01, ****p<0.0001; values are shown as mean ± standard deviation. EEV, endosomal escape vehicle; IV, intravenous; ns, not significant; PMO, phosphorodiamidate morpholino oligomer. Triceps Diaphragm Tibalis Anterior Heart Kidney Liver 0 20 40 60 80 100 Splice Correction (%) PMO654 EEV3-PMO654 0 0000 ✱✱✱✱ ✱✱✱✱ ✱✱✱✱ ✱✱✱✱ ✱✱ ns Exocyclic peptide sequence with extended linker Substitution of positively charged arginine residues with neutral charged citrullines Conjugation with PMO
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2025 Muscle Wasting 8 DUCHENNE MUSCULAR DYSTROPHY
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2025 Muscle Wasting 9 Heart Respiratory muscles Skeletal muscle Patients with Duchenne EEV-Oligonucleotide Approach >40% of patients with Duchenne3 have mutations amenable to exon skipping of exons 44, 45, 50, 51 and 53 14.0% 9.0% 8.1% 7.6% 3.8% Approximately 41,000 people in the U.S.1 and Europe2 have Duchenne SIGNIFICANT THERAPEUTIC NEED EXISTS WITHIN A VALIDATED DUCHENNE MARKET Duchenne is caused by mutations in the DMD gene, which lead to a lack of functional dystrophin, causing progressive loss of muscle function throughout the body Exon skipping therapeutics have been approved based on modest improvement in dystrophin levels ranging from ~1% to 6%4-7 1. Parent Project Muscular Dystrophy. https://www.parentprojectmd.org/about-duchenne/. Accessed August 18, 2023. 2. Europeans Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3202375. Accessed August 18, 2023. 3. Bladen, C.L. et al. Hum Mutat. 2015. 4. AMONDYS 45 PI. 5. VILTEPSO PI. 6. VYONDYS 53 PI. 7. EXONDYS 51 PI. DMD, Duchenne muscular dystrophy; EEV, endosomal escape vehicle
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2025 Muscle Wasting 10 Skeletal MuscleCardiac Muscle Wild Type Vehicle PMO-23 EEV-PMO-23 • D2-mdx mice (male, n=6-7) were treated with 4 monthly doses of either vehicle, 20 mg/kg unconjugated PMO-23 or 20 mg/kg PMO-23 equivalent of EEV-PMO-23, and the data were collected ~4 weeks after the last dose. REPEAT EEV -PMO-23 TREATMENT IN D2 -mdx MICE Robust exon 23 skipping after 4 monthly IV doses of EEV-PMO-23 in D2-mdx mice Broad dystrophin expression and restoration of muscle integrity after four monthly IV doses of EEV-PMO-23 in D2-mdx mice ****p<0.0001. Values are shown as mean ± standard deviation. D2-mdx is a DMD mouse model with a nonsense mutation in DMD exon 23 (Coley, W.D. et al. Hum. Mol. Genet. 2016). Li, X. et al. Mol. Ther. Nucleic Acids 2023. EEV, endosomal escape vehicle; DMD, Duchenne muscular dystrophy; IV, intravenous; ns, not significant; PMO, phosphorodiamidate morpholino oligomer; PMO-23, mouse DMD exon 23 skipping phosphorodiamidate morpholino oligomer. Heart Diaphragm Tibialis Anterior Triceps **** **** Vehicle PMO-23 EEV-PMO-23 0 20 40 60 80 100Exon Skipping (%) 0 20 40 60 80 100 Exon Skipping (%) 0 20 40 60 80 120 Exon Skipping (%) 100 0 20 40 60 80 100Exon Skipping (%) **** **** Vehicle PMO-23 EEV-PMO-23 **** **** Vehicle PMO-23 EEV-PMO-23 **** **** Vehicle PMO-23 EEV-PMO-23
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2025 Muscle Wasting 11 EEV -PMO PRECLINICAL STUDIES
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2025 Muscle Wasting 12 A single dose of EEV-PMO-44 produced robust human DMD exon 44 skipping and dystrophin production 2 weeks post-dose in mice amenable to exon 44 skipping EEV-PMO-44 EFFICACY IN del45hDMD. mdx MICE *p≤0.05, **p≤0.01, ***p≤0.001, ****p≤0.0001 vs. Vehicle Gastrocnemius Diaphragm Heart del45hDMD.mdx mice were treated with a single IV injection of EEV-PMO-44 (DMD exon 44 skipping EEV-oligonucleotide construct) or vehicle. Human DMD exon 44 skipping (top) and dystrophin protein expression (bottom) were analyzed in the gastrocnemius, diaphragm, and heart 2 weeks after the final dose. Percent dystrophin protein restoration is normalized to total protein and normalized to hDMD.mdx controls. Data shown as mean ± standard deviation. One-way ANOVA was used for statistical comparison; ANOVA, analysis of variance; hDMD, human dystrophin transgene; IV, intravenous. Exon 44 skipping (%)Dystrophin (%) 0 20 40 60 80 100 Vehicle 15 30 60 EEV-PMO-44 (mg/kg) 0 20 40 60 80 100 Vehicle 15 30 60 EEV-PMO-44 (mg/kg) **** **** ** **** **** ** 0 20 40 60 80 100 0 20 40 60 80 100 Vehicle 15 30 60 EEV-PMO-44 (mg/kg) Vehicle 15 30 60 EEV-PMO-44 (mg/kg) ***** **** **** * 0 10 20 40 50 60 30 0 10 20 30 40 50 Vehicle 15 30 60 EEV-PMO-44 (mg/kg) Vehicle 15 30 60 EEV-PMO-44 (mg/kg) **** **
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2025 Muscle Wasting 13 DYSTROPHIN LOCALIZATION WITH EEV -PMO-44 IN del45hDMD. mdx Mice • del45hDMD.mdx mice were treated with a single IV dose of EEV-PMO-44 or vehicle. Dystrophin protein distribution and cellular localization were analyzed by immunofluorescence in the gastrocnemius 2 weeks post-dose. Quantification via Halo Image Analysis Software is shown as the percentage of dystrophin-positive muscle fibers relative to the total number of muscle fibers as determined by laminin staining (green) and dystrophin staining (red); co-localization to the sarcolemma appears yellow and nuclei appear blue. Data shown as mean ± standard deviation. One-way ANOVA was used for statistical comparison; ANOVA, analysis of variance; hDMD, human dystrophin transgene; IV, intravenous. EEV-PMO-44 produced dose-dependent increases in dystrophin-positive muscle fibers localized to the sarcolemma of del45hDMD.mdx mice 2 weeks post-dose ***p≤0.001, ****p≤0.0001 vs. Vehicle hDMD.mdx Dystrophin/ Laminin/ Nuclei Dystrophin Vehicle 15 mg/kg EEV-PMO-44 del45hDMD.mdx Skeletal Muscle 30 mg/kg EEV-PMO-44 60 mg/kg EEV-PMO-44 0 20 40 60 80 120 100Dystrophin positive fibers (%) 15 30 60 EEV-PMO-44 (mg/kg) del45hDMD.mdx mice **** *** **** ****
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2025 Muscle Wasting 14 EEV-PMO-44 IMPROVES MUSCLE FUNCTION IN del45hDMD. mdx Mice del45hDMD.mdx mice were treated with a single IV injection of EEV -PMO-44 or vehicle. ECC-induced muscle force loss generated by repeated ECC c ontraction and tetanic force of the gastrocnemius muscle was assessed 2 weeks post-dose. Data (mean ± standard deviation) shown across 10 ECC contractions normalized into a percentage of the initial force before any ECC contrac tions and as the percentage of force retained after the 10th contraction. Vehicle - treated hDMD.mdx mice were used as a control group for normal muscle function. One -way ANOVA was used for statistical comparison to vehicle -treated del44hDMD.mdx mice. ECC, eccentric force; hDMD, human dystrophin transgene; IV, intravenous. A dose-dependent increase in resistance to membrane damage was observed following the tenth contraction, as well as an increase in tetanic force 2 weeks post-dose of EEV-PMO-44 **p<0.01, ***p<0.001, ****p<0.0001 vs. Vehicle Skeletal Muscle Membrane Stability Specific Tetanic Force 9 10 20 40 60 80 100 0 7 85 63 41 2 Number of eccentric contractions hDMD.mdx + vehicle del45hDMD.mdx + vehicle del45hDMD.mdx + 15 mg/kg EEV-PMO-44 del45hDMD.mdx + 30 mg/kg EEV-PMO-44 del45hDMD.mdx + 60 mg/kg EEV-PMO-44 % Force before injury % Force before injury (at 10th contraction) 15 30 60 EEV-PMO-44 (mg/kg) 20 40 60 80 100 **** ** **** ** del45hDMD.mdx mice 15 30 60 EEV-PMO-44 (mg/kg) 0.30 0.35 0.40 0.45 0.50 0.25 Specific tetanic force (mN-m/g) **** **** ******* del45hDMD.mdx mice
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2025 Muscle Wasting 15 CONSISTENT AND DURABLE EFFICACY OF EEV - PMO WAS DEMONSTRATED ACROSS SPECIES Significant patient benefit is implied by data in the mouse and the monkey at clinically relevant levels; in vitro data suggests much higher target engagement in patient cells Exon 44 Skipping (%) • Post IV infusion of single 45 mg/kg dose of ENTR-601-44, robust exon 44 skipping observed in biceps of treated monkeys (n=3 per cohort) for at least 12 weeks • Single IV 80 mg/kg dose of ENTR-601-44 • Tibialis Anterior Week 1 Week 2 Week 4 Week 8 Week 12 0 20 40 60 80 100 Exon 44 skipping (%) Exon 44 Skipping in hDMD Mouse Pre dose Day 2 Day 7 Day 14 Day 19 Day 28 Day 35 Day 42 Day 56 Day 70 Day 84 0 20 40 60 80 100 Exon 44 Skipping (%) Exon 44 Skipping in Monkey ~40-fold higher Healthy Cells; n=4 cells/dosePatient Cells (DMD45); n=4 cells/dose Exon 44 Skipping in Healthy and Patient- Derived Muscle Cells Untreated 3 μM 1 μM 0.3 μM 0.1 μM 0.03 μM 0 20 40 60 80 100 Exon 44 Skipping (%) ENTR-601-44 hDMD transgenic mice (left) express full-length human dystrophin gene ('t Hoen, A.C. et al. J. Biol. Chem. 2008). DMDΔ45 (right) are immortalized myoblasts from DMD patients harboring an out-of-frame exon 45 deletion and further differentiated into myotubes. Values are shown as mean ± standard deviation. ENTR-601-44 is a DMD exon 44 skipping EEV-oligonucleotide construct. DMD, Duchenne muscular dystrophy; hDMD, human Duchenne muscular dystrophy; IV, intravenous. • Robust dose-dependent exon 44 skipping was observed in DMD patient-derived muscle cells harboring an exon 44 skip-amenable mutation
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2025 Muscle Wasting 16 DELIVERY OF PMO TO SATELLITE CELLS
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2025 Muscle Wasting 17 MUSCLE SATELLITE CELLS Muscle satellite cells as a new therapeutic target with potential in several neuromuscular disorders • Quiescent satellite cells are historically challenging to access by therapeutic modalities ▪ Despite notable advancements in therapeutic strategies for DMD, current treatments primarily target dystrophic myofibers and show modest ability to generate dystrophin ▪ The lack of dystrophin leads to changes in phenotype and function of satellite cells, exacerbating disease progression by impairing muscle regeneration1 • Efficient delivery of EEV-PMO to quiescent satellite cells could enable early disease intervention ▪ EEV constructs have effectively facilitated the delivery of exon skipping PMOs to skeletal and cardiac muscle of mice ▪ Targeted correction of satellite cell dysfunction in DMD has been shown to ameliorate pathophysiology in preclinical models2 Developmental Stages of Muscle Satellite Cells3 1. Kodippili & Rudnicki Front Physiol, 2023; 2. Kwon et al. Mol Ther Meth Clin Dev, 2020; 3. Byun et al., Inflamm Regen, 2024.
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2025 Muscle Wasting 18 DISTRIBUTION OF EEV -PMO TO SATELLITE CELLS EEV-PMO efficiently co-localizes with quiescent satellite cells (Pax7 positive) at 48 hours; Qualitative data demonstrates that co-localization lasts at least 1-week post-dose • Two independent molecular techniques were utilized to determine EEV-PMO distribution within specific cell lineages across muscle tissue ▪ RNA-ISH: Highly selective and sensitive technique to assess specific cell lineages across muscle tissue (top panel) ▪ Immunohistochemistry Assessment (bottom panel) • Analysis of RNA-ISH data confirms that EEV-PMO is co- localized in 100% of satellite cells at 48 hours ▪ Quantitative assessment confirms qualitative data (data not shown) • Qualitative assessment of IHC data demonstrates co- localization of satellite cells in hDMD mice with EEV-PMO at 7 days (Top right) D2-mdx mice were treated with a single IV dose of EEV-PMO; Gastrocnemius was collected at 48 hours and analyzed by RNA-ISH. (Bottom right) hDMD mice were treated with a single IV dose of EEV-PMO; Gastrocnemius was collected at 7 days and tissues analyzed by IHC. Pax7 is a satellite cell marker (Seale, P. et al Cell 2000). hDMD, human Duchenne muscular dystrophy; IV, intravenous; RNA-ISH: RNA in situ Hybridization image analysis; IHC: immunohistochemistry. PMO Distribution (48 hours, RNA-ISH Quantitation) Pax7 (Satellite Cells)PMO Pax7 / PMO / Nuclei PMO Distribution in hDMD Mice (Day 7, IHC) Pax7 (Satellite Cells) Pax7 / PMO / NucleiPMO
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2025 Muscle Wasting 19 PMO-23 PERSISTS IN SATELLITE CELLS OF D2-mdx MICE 12 WEEKS AFTER FINAL DOSE PMO-23 co-localizes with satellite cells and newly regenerated centrally nucleated fibers 12 weeks post washout after 3 Q6W doses D2-mdx mice were treated with 3 IV doses of EEV-PMO-23 administered every six weeks; Gastrocnemius was collected 12 weeks after the 3rd dose and analyzed by immunohistochemistry. Q6W: every six weeks. D2-mdx Weeks After 1st Dose 0 6 12 24 EEV-PMO-23 EEV-PMO-23 EEV-PMO-23 Gastrocnemius muscle collected and analyzed by immunohistochemistry
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2025 Muscle Wasting 20 EEV-PMO-23 IMPROVES MUSCLE FUNCTION IN D2-mdx MICE D2-mdx mice were treated with 3 Q6W IV injections of EEV-PMO-23 or vehicle. ECC-induced muscle force loss generated by repeated ECC contraction of the gastrocnemius muscle was assessed 12 weeks after the third dose. Data (mean ± standard deviation) shown across 10 ECC contractions normalized into a percentage of the initial force before any ECC contractions and as the percentage of force retained after the 10th contraction. One-way ANOVA was used for statistical comparison. ***p<0.001, ****p<0.0001. ECC, eccentric force; IV, intravenous; med, medium; Q6W, every six weeks. Three Q6W doses of EEV-PMO (medium and high dose) maintain a significantly improved tetanic force and restored the membrane stability to wild type mice % Force before injury at 10th contraction 0 25 50 75 100 D2-mdx mice 100 % Force before injury 80 60 40 20 0 Number of eccentric contractions 9 107 85 63 41 2 Wild type + vehicle D2-mdx + vehicle D2-mdx + EEV-PMO-23 (low dose) D2-mdx + EEV-PMO-23 (med dose) D2-mdx + EEV-PMO-23 (high dose) *** ********
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2025 Muscle Wasting 21 DYSTROPHIN LOCALIZATION WITH EEV -PMO-23 IN D2 -mdx MICE • D2-mdx mice were treated with a three Q6W IV doses of EEV-PMO-23 or vehicle. Dystrophin protein distribution and cellular localization were analyzed by immunofluorescence in the gastrocnemius 12 weeks post-dose. Quantification via Halo Image Analysis Software is shown as the percentage of dystrophin-positive muscle fibers relative to the total number of muscle fibers as determined by laminin staining (green) and dystrophin staining (red); co-localization to the sarcolemma appears yellow and nuclei appear blue. Data shown as mean ± standard deviation. One-way ANOVA was used for statistical comparison. ****p≤0.0001; ANOVA, analysis of variance; hDMD, human dystrophin transgene; IV, intravenous; med, medium. Three Q6W doses of EEV-PMO-23 produced dose-dependent increases in dystrophin-positive muscle fibers localized to the sarcolemma of D2-mdx mice 12 weeks after the third dose Dystrophin/ Laminin/ Nuclei Dystrophin D2-mdx Skeletal Muscle Dystrophin-positive fibers (%) 125 100 75 50 25 0 **** **** **** D2-mdx mice High Dose EEV-PMO-23 Med Dose EEV-PMO-23 Low Dose EEV-PMO-23 Vehicle hDMD.mdx
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2025 Muscle Wasting 22 • EEV-PMO constructs demonstrated significant exon skipping and dystrophin production across several models of DMD • This suggests effective delivery of PMO therapeutics directly to muscle tissue by the EEV platform • PMO co-localizes with satellite cells and newly formed centrally nucleated fibers in skeletal muscle following EEV-PMO administration • Improved dystrophin restoration and muscle function was observed following delivery of exon-skipping PMO therapeutics to the satellite cell compartment • The ability to deliver therapeutic PMOs to muscle fibers and to quiescent satellite cells holds potential for DMD and other neuromuscular disorders SUMMARY
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2025 Muscle Wasting 23 Meet Franklin and his family, living with Duchenne muscular dystrophy