Slide 1: Corporate Presentation November 2025
[Graphic Appears Here] November 2 0 2 5 Corporate Presentation 87050-004-Part-503Nov25 13:39Page 22[Graphic Appears Here]
Slide 2: Cautionary Note Regarding Forward Looking Statements
Cautionary Note Regarding Forward Looking Statements statements This presentation regarding contains future “forward expectations, -looking plans statements” and prospects within the for the meaning company; of the the Private ability Securities to successfully Litigation achieve Reform and Act exec
Slide 3: Management team
[Graphic Appears Here] [Graphic Appears Here] Ty Howton, J.D. Gabriel Brooks, M.D. Paul Herzich Shuli Kulak, M.D. Chief Operating Officer Chief Medical Officer Chief Technology Officer Head of Corporate Strategy & Business Development [Graphic Appears Here]
Slide 4: Clinical Stage Genetic Medicines Company Targeting Neuromuscular and Cardiac Diseases
Clinical Stage Genetic Medicines Company Targeting Neuromuscular and Cardiac Diseases Research / Worldwide Program Indication Preclinical Phase 1/2 Phase 3 Discovery Rights Neuromuscular INSPIRE DUCHENNE SGT-003 Duchenne muscular dystrophy IMPACT DUCHENNE SGT-212 Friedreich’s ataxia (FA) Cardiac RYR
Slide 5: Anticipated Near-Term Milestones
Anticipated Near-Term Milestones Program Milestone Anticipated Timing Neuromuscular Interim 10 participant Phase 1/2 data (safety, microdystrophin expression & biomarker data) SGT-003 for Duchenne 30 participants dosed in INSPIRE DUCHENNE Early 2026 Anticipated meeting with FDA to discuss registrati
Slide 6: Duchenne Muscular Dystrophy
[Graphic Appears Here] D u c h e n n e M u s c u l a r D y s t r o p h y ( D u c h e n n e ) 87050-004-Part-5 03Nov25 13:39 Page 27 [Graphic Appears Here]
Slide 7: SGT-003: Next-Generation Duchenne Gene Therapy Candidate
[Graphic Appears Here] SGT Transduce -003: Next and- Generation Preserve Muscle Duchenne Gene Therapy Candidate Optimized to SGT-003 MICRODYSTROPHIN TRANSGENE SGT-003 AAV-SLB101 CAPSID Dystroglycan Binding Domain COOH NH3 Actin Binding nNOS Binding Domain Domain Unique inclusion of nNOS-binding doma
Slide 8: Duchenne is a Disease of Impaired Muscle Integrity & Dysfunction
Duchenne is a Disease of Impaired Muscle Integrity & Dysfunction1-4 In Duchenne, muscle fiber regeneration becomes impaired, leading to deterioration of muscle integrity resulting in difficulties with mobility, thoracic scoliosis, respiratory failure, and cardiac failure4 Decreased heart function Ca
Slide 9: SGT-003 optimized transgene and capsid
[Graphic Appears Here] SGT-003 AAV-SLB101 SGT-003 MICRODYSTROPHIN CAPSID TRANSGENE [Graphic Appears Here][Graphic Appears Here] SGT-003’s optimized transgene and next-generation capsid were designed with the goal of enhancing cardiac and skeletal muscle transduction and reducing liver targeting 8705
Slide 10: All participants responded to SGT-003 treatment
[Graphic Appears Here] All participants responded to SGT-003 treatment (n=10): Mean microdystrophin protein observed at consistent levels across three measures Observed comprehensive DAPC & biomarker analysis suggest coordinated downstream effect of SGT-003 Early data suggest potential cardiac benef
Slide 11: Microdystrophin positive fibers
[Graphic Appears Here] 87050-004-Part-5 03Nov25 13:39 Page 32 M[Graphic Appears Here] 87050-004-Part-5 03Nov25 13:39 Page 32 Microdystrophin positive fibers are not adjusted for fat and fibrosis; this is an absolute number. Representative images are shown. 11 1. Data cutoff of September 29, 2025. So
Slide 12: INSPIRE DUCHENNE Interim Safety Summary
INSPIRE DUCHENNE Interim Safety Summary 23 participants have received SGT-003 at ages ranging from 1 to 10 years as of October 31, 2025 [Graphic Appears Here] Eligible Age Range Ages at Enrollment Weights for Dosing Participants Enrolled Cohort (years) (years) (kg) (n) 1 4 to <7 4 to 6 ≤27.8 13 2 7
Slide 13: Liver Enzymes Remained Stable After SGT-003 Administration
Liver Enzymes Remained Stable After SGT-003 Administration SGT-003 Clinical Trial Liver Biomarkers1 (n=14) 1 ALT=alanine . Data cutoff aminotransferase; of September 29, 2025 AST=aspartate . Solid Biosciences aminotransferase; . 2. Steroid GGT=gamma tapering begins -glutamyl at Day transferase 30 pe
Slide 14: Loss of Dystrophin Leads to Progressive Degeneration of Cardiac Muscle
Loss of Dystrophin Leads to Progressive Degeneration of Cardiac Muscle1 Cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy2 Cardiac disease 25% 59% 98% 40% is underway long before overt cardiac evidence of evidence of evidence of of these patients dysfunction appears4 cardiomy
Slide 15: Stable-to-Improved Cardiac Function Observed After SGT-003 Dosing
Stable-to-Improved Cardiac Function Observed After SGT-003 Dosing Early observations of improved cardiac function driven by participants with low to low-normal baseline left ventricular ejection fraction (LVEF)1 Participants who Participants who Participants who reached Day 90 reached Day 180 reache
Slide 16: Troponin Reductions May Indicate Early Signals of Cardiac Treatment Effect
Troponin Treatment Reductions Effect May Indicate Early Signals of SGT-003 Cardiac Troponin I is released during myocardial cell injury, acting as a signal of muscle breakdown and a surrogate for cardiac myocyte damage1 INSPIRE DUCHENNE Percent Change From Baseline Troponin2 80 Change 40 % 0 Mean -4
Slide 17: Dystrophin Organizes Critical Skeletal and Cardiac Muscle Proteins
for Dystrophin Muscle Health Organizes Critical Skeletal and Cardiac Muscle Proteins Required OVERVIEW OF THE SKELETAL DAPC OVERVIEW OF THE CARDIAC DAPC The absence of dystrophin can cause dissolution, or significant downregulation, of the entire DAPC and many of the mechanoprotective and mechanotra
Slide 18: Microdystrophin Expression Observed at Day 90 Post SGT-003 Treatment
Compelling Treatment Microdystrophin Expression Observed at Day 90 Post SGT-003 Comprehensive orthogonal measurements showed consistent microdystrophin expression EXAMPLE MICRODYSTROPHIN BIOPSY1 1 measured . Data cutoff by IF of. September Dystrophin 29, positive 2025 fibers . Solid are Biosciences
Slide 19: β-sarcoglycan and nNOS are Key Components of the DAPC
â-sarcoglycan and nNOS are Key Components of the DAPC SARCOGLYCAN COMPLEX FORMATION1 MICRODYSTROPHIN CONTRUCTS WITHOUT R16/R17 CANNOT RECRUIT nNOS4 â-sarcoglycan tightly associates with ä-sarcoglycan to form a functional core that recruits ã- and á-sarcoglycan2 Disruption of the â/ä core interferes
Slide 20: Restoration of Key Elements of the DAPC After SGT-003 Treatment
Restoration of Key Elements of the DAPC Observed After SGT-003 Treatment MICRODYSTROPHIN POSITIVE FIBERS â POSITIVE -SARCOGLYCAN FIBERS MICRODYSTROPHIN â-SARCOGLYCAN + nNOS ACTIVITY Positive Fibers SGT-003 microdystrophin positive fibers (%) achieved a statistical correlation with both â-sarcoglycan
Slide 21: eMHC Expression Is Upregulated in Dystrophic Muscle Fibers
eMHC Expression Is Significantly Upregulated in Dystrophic Muscle Fibers1-3 Muscle stem cells (satellite cells) are activated to repair and replace damaged muscle fibers–during this process, newly formed muscle fibers transiently express embryonic myosin heavy chain (eMHC)3,4 Ultimately, muscle fail
Slide 22: Muscle Biopsy Scans Showed Uniform Improvements in eMHC
Full Slide Scans of Muscle Biopsy Sections Showed Uniform Improvements in eMHC1,2 MICRODYSTROPHIN POSITIVE FIBERS eMHC FIBERS POSITIVE MICRODYSTROPHIN + eMHC 22 Data cutoff of September 29, 2025. Solid Biosciences. Representative images from the same participant are shown. © 2025 Solid Biosciences
Slide 23: Comprehensive Reductions in Muscle Injury Signals After SGT-003
Muscle Comprehensive Integrity Reductions After SGT-003 in Muscle Treatment Injury Signals Suggest Improved Improved muscle integrity may support slower disease progression and better long-term clinical outcomes1-3 DETERIORATION OF MUSCLE INTEGRITY IN DUCHENNE OBSERVED IMPROVEMENTS IN MUSCLE INTEGRI
Slide 24: Reductions in CK Levels Observed After SGT-003 Treatment
Reductions in CK Levels Observed After SGT-003 Treatment Reductions in CK are signals of improved muscle integrity; CK is released from muscle fibers as a result of muscle damage1 INSPIRE DUCHENNE Percent Change From Baseline CK2 CK=creatinine kinase; LS=least squares. 24 1. Oshida N, et al. Sci Rep
Slide 25: Improvements in Measures of Muscle Integrity & Resilience
SGT Improvements -003 Treatment in Measures of Muscle Integrity & Resilience Observed After Comprehensive and thorough assessment of muscle injury and metabolism SERUM ALT Percent Change SERUM AST Percent Change SERUM Titin Percent Change SERUM LDH Percent Change From Baseline1 From Baseline1 From B
Slide 26: Promising Clinical Profile Positions SGT-003 for Potential Functional Benefit
Promising Clinical Profile Positions SGT-003 for Potential Functional Benefit Restoration of the Potential Optimized Microdystrophin Enhanced Dystrophin-Associated Functional Transduction Expression Muscle Integrity Protein Complex Outcomes Western Mass Serum/histologic Time to rise PCR blot spec Im
Slide 27: Neuromuscular Pipeline Program: Friedreich's Ataxia (FA)
Neuromuscular Pipeline Program F r i e d r e i c h ‘ s A t a x i a ( F A ) © 2025 Solid Biosciences
Slide 28: Friedreich's Ataxia: Progressive Genetic Neuromuscular Disease with High Unmet Need
Friedreich’s A Progressive Ataxia Genetic (FA): Neuromuscular Disease with High Unmet Medical Need Cause FA is a monogenic disease resulting from a deficiency of the frataxin (FXN) protein, which is important for mitochondrial function. Affected Population Postulated Mechanism: Decreased levels of F
Slide 29: Introducing SGT-212: Dual Administration Approach for FA
Introducing Both Neurologic SGT -and 212: Cardiac The Only Manifestations Dual Administration of FA Approach to Address Intravenous (IV) Administration Direct Dentate Nuclei (IDN) Infusions* • Focused on treating largest cause of • Removes challenges of crossing blood-brain barrier mortality in Frie
Slide 30: SGT-212 Administration Resulted in Neurological and Neuromotor Improvements
SGT and -Neuromotor 212 Systemic Function Administration Improvements Resulted in Significant Neurological Neuronal proof-of-concept achieved in disease-relevant knockout mouse model (nKO) Neurological Assessment Score Neuroscore1 (Day 56) Neuromotor Function Assessment RotaRod2 (Day 60) Asterisks=s
Slide 31: SGT-212 Systemic Administration Demonstrated Resolution of Cardiac Phenotype
Expression, SGT-212 Systemic Activity Administration and Resolution Demonstrated of Cardiomyopathy Cardiac Phenotype FXN Cardiac proof-of-concept achieved in disease-relevant knockout mouse model (cKO) FXN Cardiac Expression Mitochondrial Function Indicator of Cardiac Structure In Situ Hybridization
Slide 32: IDN Administration of SGT-212 in NHPs Resulted in Robust FXN Expression
IDN the Cerebellum Administration in NHPs of SGT at -Clinically 212 Resulted Relevant in Robust Dose FXN Expression in hFXN Expression in Dentate Nuclei (Cerebellum) In Situ Hybridization* ISH hFXN NHP #1 NHP #2 NHP #3 NHP #4 Day 28 Day 28 Day 28 Day 90 hFXN Properly Localized to Dentate Nuclei (Cer
Slide 33: Solid Has Built Understanding and Expertise in FA
Solid Extensive has Built Preclinical Robust Work Understanding in NHPs and Expertise in FA Through [Graphic Appears Here] Substantial in-house preclinical work and preclinical studies by collaborators have been conducted across multiple candidates, routes of administration & dose levels Overall NHP
Slide 34: FALCON Clinical Trial Design: SGT-212 Phase 1b Study
FALCON Clinical Trial Design: SGT-212 Phase 1b Study First-in-Human, Open-Label, Multi-Center Study to Enroll a Minimum of 6 Participants [Graphic Appears Here] To evaluate the safety and tolerability of IDN infusion and systemic IV infusion of SGT-212 gene therapy in participants with FA Explorator
Slide 35: Cardiac Lead Program: CPVT
Cardiac Lead Program C a t e c h o l a m i n e r g i c P o l y m o r p h i c V e n t r i c u l a r T a c h y c a r d i a ( C P V T ) © 2025 Solid Biosciences
Slide 36: Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
a Catecholaminergic Fatal Disorder in a Polymorphic Young Population Ventricular Tachycardia (CPVT): Cause CASQ2 & RYR2 proteins: Regulate cardiac calcium (Ca2+ ), important for electrical conduction and cardiac contraction / relaxation Affected Population Postulated Mechanism: Mutations in RYR2 or
Slide 37: CPVT Represents High Unmet Need With No Approved Therapies
CPVT Underlying Represents Cause High of Disease Unmet Need With No Approved Therapies That Treat [Graphic Appears Here] [Graphic Appears Here] [Graphic Appears Here] [Graphic Appears Here] CPVT is a Mutations in RYR2 Altered calcium ion channelopathy; a (calcium channel) and channels impact electri
Slide 38: Rationale for CASQ2 Augmentation in RYR2 CPVT
Rationale for CASQ2 Augmentation in RYR2 CPVT In RYR2 pathogenic mutations, normal CASQ2 levels are insufficient to maintain RYR2 in a closed conformation during diastole in high calcium flux states (such as with adrenaline) RYR2 Mutation-Related CPVT Arrhythmia Mutations in RYR2 make the channel mo
Slide 39: Rationale for CASQ2 Augmentation in RYR2 CPVT (cont.)
Rationale for CASQ2 Augmentation in RYR2 CPVT (cont.) Cardiac delivery of SGT-501 is intended to increase CASQ2, thus enhancing Ca2+ buffering and counteracting Ca2+ sensitivity caused by RYR2 pathogenic mutations RYR2 Mutation-Related CPVT + Increased CASQ2 expression Normal Rhythm Increased CASQ2
Slide 40: AAV Delivery of Human CASQ2 Used to Support CPVT Proof-of-Concept
AAV RYR2 Gene CPVT Delivery Transgenic of Human Mouse CASQ2 Model Used To Support Proof-of-Concept For ECG response to â-adrenergic stimulation in WT and RYR2 transgenic mice 85 days post vehicle or SGT-501 treatment Type Normal heart rhythm WT Mice in WT background Dosed With Vehicle Wild strain an
Slide 41: SGT-501 Elicited Steady Cardiac Protein Expression in Mice and NHPs
SGT-501 Elicited Steady Cardiac Protein Expression in Mice and NHPs Clinically relevant expression levels continued through month 6 indicating potential durability and stability of expression hCASQ2 Protein Expression Mouse Kinetics: Expression significantly increased until Day 56, followed by conti
Slide 42: SGT-501 Demonstrated Protection From Sustained VT & Arrhythmia
SGT-501 Demonstrated Protection From Sustained VT & Arrhythmia SGT-501 demonstrated dose-responsive reduction in adrenaline-mediated VT in RYR2 adult mice Treatment Efficacy (Prevention of Arrhythmia) ***P=0.0002 **P=0.017 Proof-of-Concept Study Efficacy 75 **P=0.0035 ns SGT-501 treatment resulted i
Slide 43: SGT-501 was Well Tolerated in NHP GLP Toxicology Study
SGT-501 was Well Tolerated in NHP GLP Toxicology Study FINDINGS • SGT-501 was well tolerated at each evaluated dose level: NHP GLP Tox Study no adverse effects were observed on hematology or serum clinical chemistry in NHPs after treatment. 3- and 6-month timepoints 6 treatment groups across 3 dose
Slide 44: ARTEMIS Clinical Trial Design: SGT-501 Phase 1b Study
ARTEMIS Clinical Trial Design: SGT-501 Phase 1b Study First-in-Human, Open-Label, Multi-Center Study to Enroll a Minimum of 6 Participants Activation of first clinical trial site expected Q4 2025 Objective Design Endpoints Primary Objective Design Primary Endpoint • To evaluate the safety and tolera
Slide 45: Contact
500 Rutherford Avenue, Third Floor, Charlestown, MA 02129 investors@solidbio.com www.solidbio.com Learn about ways to partner with us: www.solidbio.com/partners/ © 2025 Solid Biosciences