Income Statement Presentation 2023

SRP-9001 in DMD: Pivotal Ph III results expected at year-end First and potential best-in-class gene therapy Delandistrogene moxeparvovec SAREPTA Roche THERAPEUTICS Clinical trial program overview ITR ssDNA C Promoter Transgene PolyAt 5' 3' OH ITR* Study DMD subgroup Phl Ph II Ph III Comment 101 Ambulatory, 4-7 yrs. US filing by Sarepta Important determinant of 102 expression levels and specificity 10,11 Important determinant of functional impact 10.12 Ambulatory, 4-7 yrs. Delandistrogene moxeparvovec Expression MHCK7 Function Micro-dystrophin Vector H One of the important determinants of safety experience and transduction efficiency Safety AAVrh74 103 (ENDEAVOR) 301 (EMBARK) 302 (ENVOL) 303 (ENVISION) Ambulatory, 3-18 yrs Non-ambulatory, all ages Ambulatory, 4-7 yrs. Ambulatory, 0-3 yrs. US filing by Sarepta US filing by Sarepta* Data read out expected Q4 2023 Ph II to initiate in 2023 Ambulatory, 8-18 yrs Non-ambulatory, all ages Ph III to initiate in 2023 • Targeted delivery of functional shortened dystrophin transgene to muscle tissue Vector and promotor specifically designed for skeletal & cardiac muscles Transgene can enable meaningful and durable response • ● Positive functional and clinically meaningful results at multiple time points (including 1, 2, 4 years after treatment) with consistent safety profile shown for >80 patients US filing by partner Sarepta accepted and priority review granted with PDUFA date set for 29th May ⚫ Ph III (EMBARK) read out expected in Q4; results will form the basis for EU filing . DMD-Duchenne muscular dystrophy; PDUFA-prescription drug user fee act; Delandistrogene moxeparvovec in collaboration with Sarepta Therapeutics; *Cohort 1 only 31

View entire presentation