Director, Biostatistics

Why Sarepta? Why Now? The promise of genetic medicine has arrived, and Sarepta is at the forefront. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. In 2023, we launched our fourth therapy and the first ever gene therapy to treat Duchenne. Why Sarepta? Why Now? The promise of genetic medicine has arrived, and Sarepta is at the forefront. We hold a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. In 2023, we launched our fourth therapy and the first ever gene therapy to treat Duchenne. We’re looking for people who see unlimited potential in themselves and who are motivated by an unwavering commitment to patients. What Sarepta Offers At Sarepta, we care deeply about all the people in our community and believe in the importance of supporting them in all aspects of their lives. We aspire to maintain a culture that acknowledges people bring their whole selves to work, and we will strive to help everyone in our community integrate their work and personal lives while maintaining productivity. We are committed to offering a range of benefits and work-life resources designed to support people in the following areas: Physical and Emotional Wellness Financial Wellness Support for Caregivers For a full list of our comprehensive benefits, see our website: The Importance of the Role The person in this position will be working with a cross-functional team as the project biostatistician responsible for statistical activities at the compound level or study level in DMD or siRNA platforms. Beyond traditional SAP-driven deliverables, this role calls for a hands-on analyst who can explore data deeply, partner with clinical development to shape scientific questions, and deliver rapid insights using modern analytical tools. It is an excellent opportunity to work at the forefront of precision genetic medicine, making an impact on the development of transformative therapies that may change the lives of patients. The ideal candidate is a good communicator, high-energy, self-motivated, and forward thinking. Experience working in rare disease is desirable, but not required. The Opportunity to Make a Difference Lead collaboration with internal and external (e.g., CRO) team members to coordinate the planning and execution of statistical deliverables Lead/conduct statistical modeling to enable robust and efficient statistical designs and to address identified or potential statistical issues arising in studies or programs Contribute to clinical protocol development, including authoring of the Statistics section and reviewing of other sections by applying statistical principles Author or review statistical analysis plans for clinical trials and for integrated summaries of safety/effectiveness (ISS/ISE), and author or oversee the development of shells for tables, figures and listings Review case report form (CRF) designs to ensure data collection meets the requirements of statistical analyses Design and specify randomization schedules; review and approve test randomization lists Provide statistical input to data monitoring committee (DMC) charters, independent review charters, and other study-level documents Review analysis dataset specifications Perform QC/QA of statistical deliverables including validation of key analysis results Perform ad hoc and exploratory statistical analyses as needed Contribute to clinical study reports, including authoring of statistical methods and interpretation of the study results Be accountable for assigned statistical activities in support of IND/NDA/MAA or other regulatory submissions Address statistical questions/comments from FDA and other regulatory agencies, and review and address comments by IRB/ECs Support and contribute to the preparation of publications, including manuscripts, posters and oral presentations Contribute or lead standardization and process improvement efforts for Biostatistics and contribute to cross-functional process improvement efforts Represent Sarepta regarding statistical issues in meetings with external parties including regulatory agencies and corporate partners Serve as a mentor to junior team members Conduct hands-on exploratory analyses beyond pre-specified SAP deliverables, probing unexpected results, testing sensitivity scenarios, and generating rapid insights to inform program decisions Partner proactively with Clinical, Medical, and Regulatory colleagues to identify and prioritize scientific questions that warrant exploratory investigation, bringing Biometrics perspective early, not only at the analysis stage Leverage modern analytical tools (R, Python, interactive visualization apps) alongside SAS to accelerate data-to-insight timelines, while maintaining clear documentation and traceability of exploratory work Contribute to fit-for-purpose validation practices, distinguishing between outputs intended for internal learning versus those that require production-grade validation for external communication or regulatory use More About You Ph.D. (strongly preferred) with 9+ years or Master’s degree with 10+ years of experience in statistics or related discipline in the pharmaceutical or biotech industry Demonstrated ability and experience in the design, analysis, and reporting of clinical trials Experience in NDAs, MAAs or other regulatory submissions preferred In-depth knowledge of statistical methods for clinical trials, including both frequentist and Bayesian approaches In-depth knowledge of FDA, EMA, and ICH regulations and guidelines Knowledge of gene therapy, rare disease, or neuromuscular diseases is desirable, but not required Proficient in statistical programming in SAS and R; Python and other languages a plus Proficient in statistical design software such as EAST Ability to concurrently lead statistical efforts for multiple studies Understanding of data standards, including SDTM and ADaM Ability to oversee statistical services provided by CROs and/or contractors Ability to work independently, guide team and junior biostatisticians, and act with initiative to address issues Ability to collaborate effectively with colleagues from all other functions Excellent written and oral communication skills, including the ability to effectively communicate statistical concepts in a clear and concise manner Familiarity with external control arms, real-world evidence, or observational study methods is a plus Willingness and ability to perform hands-on data exploration using R and/or Python, in addition to SAS Enthusiasm for learning and harnessing modern AI tools and workflows to enhance analytical productivity and scientific insight Demonstrated experience partnering with clinical development teams to shape analytical strategy, not just execute pre-defined plans #J-18808-Ljbffr Sarepta Therapeutics