Why Sarepta? Why Now?

The promise of genetic medicine has arrived, and Sarepta is at the forefront. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development in gene therapy, RNA and gene editing. 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.

The Importance of the Role
 

The summer intern will be working with talented and highly experienced biostatisticians. They will have the opportunity to work closely with project teams and develop knowledge about clinical trial and real-world study designs, and research cutting-edge analysis strategies incorporating machine learning (ML) and artificial intelligence (AI). The project focus will be on how to efficiently quantify treatment benefits offered by products in Sarepta’s portfolio in clinical trials as well as real world settings. The goal will be to identify a scientifically sound method that maximizes the probability of regulatory success and fits commercial needs. Statistical methods combined ML / AI will be evaluated in their ability to infer causal effects in a rare disease setting efficiently. Statistical properties as well as clinical interpretability and suitability with the company’s regulatory and commercial strategy will be evaluated. The intern will build upon and continue the research started last summer on the use of causal ML methods in rare disease setting. Extensions may include researching the use of ML / AI to generate synthetic data or digital twins to supplement the data from a clinical trial. It is expected that the work will lead to internal recommendations and scientific communications that have far-ranging impact for the rare disease community. It will also provide opportunities for the intern to showcase their abilities and hone new techniques and skills.

The Opportunity to Make a Difference

• The intern will be responsible for developing understanding of the company’s data on key efficacy measurements, perform analysis of repeated measures data and time to event data. 

• The intern will perform statistical simulations in SAS, R, Python to assess different statistical methods/models and their performance. 

• Perform ad-hoc and exploratory statistical analyses as needed.

• Support and contribute to the preparation of publications, including manuscripts, posters, and oral presentations.

More about You

• MS or equivalent in statistics, biostatistics or related field, preferably a PhD candidate

• Proficiency in statistical methods, including generalized linear mixed models, survival analysis.

• Ability to handle large data in SAS and R and perform statistical simulations

• Knowledge and hands-on experience with causal inference, machine learning, artificial intelligence (e.g., General Adversarial Networks, Transformers, Bayesian Networks), survival analysis, propensity score methods, external control comparison, Bayesian methods desirable

• Excellent communication and presentation skills, including the ability to effectively communicate statistical and data science concepts in a clear and concise manner to non-technical audience. 

• Good organizational skills, sufficient to multi-task in a fast-paced environment with changing priorities.

• Team player, with ability to collaborate successfully across functions.

• Candidates must be legally allowed and available to work full-time for up to 12 weeks in the summer of 2025

Program Timeline:

This application is for a 12-week summer internship program that will start on May 19th and conclude on August 8th, 2025.

What Now?

We’re always looking for solution-oriented, critical thinkers.

So, if you’re comfortable with ambiguity and candor, relish challenging yourself, and place kindness and integrity at the forefront of how you approach your peers and work, then we encourage you to apply.
 

#LI-Remote#LI-MC1This position is remote. However, 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.The targeted salary range for this position is $21 - $25 per hour depending upon years of education completed and nature of role.Candidates must be authorized to work in the U.S.Sarepta Therapeutics offers a competitive compensation and benefit package.Sarepta Therapeutics is an Equal Opportunity/Affirmative Action employer and participates in e-Verify.