Director, Biostatistics

Why Sarepta?

Sarepta is at the forefront of genetic medicine, holding a leadership position in Duchenne muscular dystrophy (Duchenne) and building a robust portfolio across muscle, central nervous system, and cardiac diseases. In 2023, Sarepta launched its fourth therapy and the first-ever gene therapy to treat Duchenne. We seek individuals who see unlimited potential in themselves and are motivated by an unwavering commitment to patients.

What Sarepta Offers

Sarepta is committed to supporting employees in all aspects of their lives, fostering a culture where people bring their whole selves to work. We offer a range of benefits and work-life resources, including:

• Physical and Emotional Wellness
• Financial Wellness
• Support for Caregivers

For a full list of comprehensive benefits, visit our website.

The Importance of the Role

The person in this position will serve as the project biostatistician, working with a cross-functional team on statistical activities at the compound or study level for DMD or siRNA platforms. This role requires 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. The ideal candidate is a strong communicator, high-energy, self-motivated, and forward-thinking. Experience in rare disease is desirable but not required.

Responsibilities

• Lead collaboration with internal and external (e.g., CRO) team members to coordinate the planning and execution of statistical deliverables.
• Lead or conduct statistical modeling to enable robust and efficient statistical designs and address identified or potential statistical issues in studies or programs.
• Contribute to clinical protocol development, including authoring the Statistics section and reviewing other sections by applying statistical principles.
• Author or review statistical analysis plans for clinical trials and 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 statistical methods and interpreting 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 cross-functional teams.
• 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 warranting exploratory investigation, bringing Biometrics perspective early.
• Leverage modern analytical tools (R, Python, interactive visualization apps) alongside SAS to accelerate data-to-insight timelines while maintaining clear documentation and traceability.
• Contribute to fit-for-purpose validation practices, distinguishing between outputs for internal learning versus those requiring production-grade validation for external communication or regulatory use.

Requirements

• Ph.D. (strongly preferred) with 9+ years or Master’s degree with 10+ years of experience in statistics or a 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 teams and junior biostatisticians, and act with initiative to address issues.
• Ability to collaborate effectively with colleagues from all functions.
• Excellent written and oral communication skills, including the ability to effectively communicate statistical concepts clearly and concisely.
• 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.

Schedule

This position is hybrid. You will be expected to work on-site at one of Sarepta’s facilities in the United States and/or attend Company-sponsored in-person events from time to time.

Pay

The targeted salary range for this position is $206,000 - $257,500 per year. The salary offer is commensurate with Sarepta’s compensation philosophy and considers factors including education, training, experience, external market conditions, criticality of role, and internal equity.

Candidates must be authorized to work in the U.S.