Stephen Baine, Ph.D.

• Led strategic direction for more than six concurrent nonclinical programs across gene therapy, gene editing, and siRNA platforms with oversight of study design, regulatory strategy, budget directives, and team leadership. • Developed nonclinical executive summaries and risk matrices for multiple preclinical programs (siRNA, AAV gene therapy, gene editing) and presented strategic recommendations to senior management and executive committees. • Engineered AAV gene therapy platform including in vivo and in vitro potency systems, vector design optimization, and transgene/promoter efficacy evaluations across multiple therapeutic modalities. • Crafted critical regulatory documentation for pre-IND, IND, and BLA submissions including briefing books, investigator brochures, integrated development plans, and development safety update reports. • Cultivated and mentored team of Ph.D.-level scientists while directing efficient advancement of internal gene therapy assets from discovery through clinical development. • Presented Sarepta's gene therapy programs through oral presentations at ASGCT 2024 and 2025 to global scientific audiences, including selected oral presentations on SRP-9003/5 preclinical data • Founded and directed Cardiac Innovation Research Team (IRT), establishing strategy for novel cardiac gene therapy and editing programs.

• Served as nonclinical lead for global limb girdle muscular dystrophy gene therapy portfolio, architecting strategic roadmaps for IND-enabling studies and regulatory submissions. • Represented nonclinical sciences for Sarepta global program teams across gene therapy and gene editing platforms with senior leadership, clinical teams, and external regulatory agencies. • Leveraged subject matter expertise in AAV vector technologies including design, cloning, transfection, and cell-based screening systems for transgene efficacy optimization. • Documented FDA responses, EMA scientific advice briefings, study protocols, and preclinical study reports conveying program status internally and to regulatory agencies. • Evaluated business development opportunities for gene therapy partnerships, assessing competitive landscape and delivering strategic recommendations.

• • Directed nonclinical strategy for LGMD sarcoglycan portfolio, formulating approaches for PD efficacy studies, GLP toxicology programs, and potency assay development. • Coordinated business development activities evaluating external gene therapy and gene editing opportunities with strategic fit assessments. • Engineered and executed bioanalytical and tissue-based assays for pharmacokinetic and pharmacodynamic characterization of gene therapy candidates.

• Guided preclinical pharmacodynamic efficacy, GLP toxicology, and potency programs within LGMD gene therapy pipeline. • Optimized and validated in vivo functional assays for pharmacodynamic studies in large and small animal models. • Investigated disease pathogenesis and established biomarkers using qPCR, ddPCR, Western blot, and immunofluorescence methods.

• Identified signaling mechanisms regulating cholinergic signaling in heart failure using Western blot, qPCR, proximity ligation assay, and confocal microscopy techniques. • Conducted in vivo and in vitro pharmacology experiments using echocardiography, electrocardiograms, cardiomyocyte isolation, and single-cell electrophysiology in preclinical models. • Published findings in peer-reviewed journals and presented at national and international conferences.

-Produce preclinical and clinical lots of of material in accordance with manufacturing batch records and approved standard operating procedures while adhering to all applicable regulations and standards (i.e GMP, GTP, AABB) -Recognize, monitor, and evaluate technical instrument performance or problems. -Perform and analyze simple as well as complex procedures blood products intended for administration into patients for immuno-therapeutic purposes.

- Managed inventory responsibilities of consumable materials for Manufacturing Department via implementation of spreadsheets using Microsoft Excel software. - Maintain Manufacturing batch record files including check-in and check-out procedures for quality assurance and manufacturing personnel. -Prepared and formulated reagents within a class 10,000 clean room environment. -Documented and serviced equipment used in a clean room and restricted access manufacturing area.