Slide 1: AAV-SLB101: A Next-Generation Rationally Designed Capsid Demonstrates Highly Potent Cardiac Tropism
AAV-SLB101: A Next-Generation Rationally Designed Capsid Demonstrates Highly Potent Cardiac Tropism and Initial Clinical Safety Jessica F Boehler, Patrick Gonzalez, Jamie Marshall, Tiffany Willacy, Irene Larena, Meghan Soustek-Kramer, Marie Ralff, Glen B Banks, Matthew Harmelink, Jessie Hanrahan, Pa
Slide 2: Forward Looking Statement
Forward Looking Statement This presentation contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the company; the ability to successfully achieve and execute on th
Slide 3: Acknowledgments
Acknowledgments CVCT and Overcome • Faiez Zannad, Patrick Wahby, Javed Butler, Silvia Priori, Roger Hajjar DMD Community • PPMD, Duchenne UK, TREAT-NMD, MDA, Jett Foundation Solid Biosciences • Preclinical and clinical development departments 3 © 2025 Solid Biosciences - Nonconfidential
Slide 4: SGT-003 Is Solid's Next-Generation, Investigational Microdystrophin Gene Transfer Therapy That Uses AAV-SLB101
SGT-003 Is Solid’s Next-Generation, Investigational Microdystrophin Gene Transfer Therapy That Uses AAV-SLB101 AAV-SLB101 is a proprietary, rationally designed muscle-tropic capsid used in Solid Biosciences’ investigational gene therapy, SGT-003 SGT-003 is currently being evaluated in the INSPIRE DU
Slide 5: AAV-SLB101 Showed Improved Muscle Transduction and Lower Liver Distribution Compared With AAV9
AAV-SLB101 Showed Improved Muscle Transduction AAV-SLB101 MICRODYSTROPHIN TRANSGENE and Lower Liver Distribution Compared With AAV9 mdx mice DAY 0 DAY 30 Head-to-Head Comparison of Biodistribution in mdx Mouse Model of Duchennea AAV-SLB101 AAV9 QUADRICEPS HEART LIVER 4x105 2 3.00 5 6x10 26 1x10 ****
Slide 6: AAV-SLB101 Resulted in Higher Biodistribution and Expression Compared With AAVrh74
1x1014 vg/kg AAV-SLB101 Resulted in Higher Biodistribution MICRODYSTROPHIN AAV-SLB101 or AAVrh74 TRANSGENE and Expression Compared With AAVrh74 mdx mice DAY 0 DAY 30 Head-to-Head Comparison in mdx Mouse Model of Duchenne Immunofluorescence BIODISTRIBUTION MICRODYSTROPHIN QUADRICEPS EXPRESSION (WB) A
Slide 7: AAV-SLB101 Transduction Efficiency Was Maintained in NHP Studies and Human Cell Lines
AAV-SLB101 Transduction Efficiency Was Maintained in NHP Studies and Human Cell Lines Compared With Initial Results in Mice Transduction Efficiency of AAV-SLB101 Compared With AAV9 in NHPs and Human Cell Models 5x1013 vg/kg AAV-SLB101 LUCIFERASE NHP TRANSGENE IN VITRO CELLULAR MODELS or AAV9 DAY 0 D
Slide 8: INSPIRE DUCHENNE Clinical Study of SGT-003: AAV-SLB101-CK8-Microdystrophin
INSPIRE DUCHENNE Clinical Study of SGT-003: AAV-SLB101-CK8-Microdystrophin 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 to <12 7 to 10 ≤39.7 8 3 0 to <4 1 to 3 ≤17.0 2 Total 0 to <12 1 to 10 ≤39.7 23 SGT-
Slide 9: Microdystrophin Protein, Data From INSPIRE DUCHENNE Clinical Trial of SGT-003
Data From INSPIRE DUCHENNE AAV-SLB101-CK8-MICRODYSTROPHIN Clinical Trial of SGT-003 Duchenne participants DAY 0 DAY 90 DAY 360 Vector Genome Copies/Nucleus Day 90 Day 360 Example Microdystrophin Biopsy Dose (n=10) (n=2) 1x1014 vg/kg 13 12 Baseline SGT-003 Microdystrophin Expression Participants who
Slide 10: Reassuring-to-Promising Cardiac Function After SGT-003 Dosing
Reassuring-to-Promising Cardiac Function After SGT-003 Dosing1 ABSOLUTE LVEF OVER TIME (%) ABSOLUTE CHANGE FROM BASELINE LVEF (%) 60%-69% normal range of LVEF = mean ± SD2,a Improved cardiac function was observed in participants 85 18 Mean Absolute LVEF, % (n=14 at baseline) with low to low-normal L
Slide 11: AAV-SLB101 Overcomes the Challenges of First-Generation Capsids
AAV-SLB101 Overcomes the Challenges of First-Generation Capsids Higher biodistribution and transgene expression were achieved with AAV-SLB101 compared with AAV9 in animal (mdx mice, NHPs) and human cell models in key tissues for Duchenne, including skeletal muscle and heart Reductions in AAV-SLB101