Objective: The Strategic CMO drives clinical and product development strategy supporting commercialization and impacting corporate valuation through key internal and external stakeholder and investor interactions. Clinical Development designs and maintains currency of all studies; works cross‐functionally to incorporate and balance regulatory requirements, payer data needs, and operational constraints in study designs. Clinical Operations operationalizes study designs for data generation to support registrational submissions. Medical Affairs focuses on data analysis, distillation, and dissemination so all stakeholders get the most current and accurate information on safety, efficacy, method of action, and differentiation of the company’s products. Challenge/problem: An experienced medical professional becomes CMO at a pre‐IPO gene therapy company – an exciting opportunity with challenges. How does the CMO remain strategic and prevent relegation – addressing urgent operational issues instead of building out a well thought‐through medical office? The CMO leveraged external partners focused on Medical Affairs, Pharmacovigilance and Clinical Development to help build the function and execute the strategy. Solution: Start Lean. Build a scalable framework. Expand with triggers. Outcome: Hire effectively to focus on value generating pieces. Leverage contractors and consultants for short‐term SME needs. Develop growth plans, models, and strategic frameworks. Identify potential new hires in advance to prepare for growth. Driven by corporate, regulatory, and competitive factors, expand the medical organization accordingly. Switch contractors and consultants to FTE’s once the role is proven valuable and needed. Benefits: One of the most successful newly launched companies, on its way towards becoming a leading gene therapy company. The Strategic CMO drives product research and development strategy and provides key support in external stakeholder and investor interactions Data / Evidence Generation Confidence Generation Clin Dev Clin Ops Med Affairs Traditional Functions Bridge Roles Strategic Outcomes Pre‐clinical • Site Setup • CRO Management • GCP • Medical Writing • KOL Management • Patient Advocacy Engagement • Clinician Education / Pubs • Scientific Exchange • HEOR • Clinical <‐‐> Investor Relations/CEO • Clinical <‐‐> Commercial • Study Design • Clinical Research • Biostats Phases 1‐3 Pre‐Launch Submission & Approval Launch IND Expansion • Robust and flexible clinical development plan • Strong patient advocacy based on science • Compliant sites and operations • Scientific <‐‐> Commercial • Patient/Site/Clinician <‐‐> Company • Readiness against competitive factors • Key contributions to investment community stakeholders • Strong demand for company success from investor community – based on the data    Clinical Development designs and maintains currency of all studies; works with Clinical Ops, Regulatory and Commercial operations to incorporate any operational constraints and feasibility aspects Clinical Development Study Design Creation Biostatistics Clinical Research Operational Collateral Review Scientific Data Review Pre‐clinical to Medical Bridge Study Design Education • Study synopsis • Study protocol • Statistical Analysis Plan • Scenario planning based on external factors • Regulatory submissions • Competitor analysis Clinical Operations operationalizes the study design and is responsible for data generation to support registrational data submissions Clinical Operations Study Design Input Site Activation CRO Manage‐ ment Supporting Vendor Management Compliance and Assurance Data Manage‐ ment Contrac‐ ting Coordina‐ tion • SOPs and procedural documentation • Trial‐related collateral • Clinical data sets • Execution of studies based on timelines Medical Affairs focuses on data analysis, distillation, and dissemination to ensure external stakeholders get the most up to date and accurate information on the safety, efficacy, m