Year: 2020-21

Company: Sarepta Therapeutics

Liaison(s): Luca Maggioni, Emily Regan, Allicyn Aubut

Sarepta Therapeutics Inc. is a global biotechnology company on an urgent mission: to engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. Based in Cambridge, MA, Sarepta is ushering in a new era of drug development with the goal of to drive efficiencies, including shortening the time from lab to patient. Sarepta has over 40 therapies in various stages of development that utilize various modalities including gene therapy to treat diseases such as Duchenne Muscular Dystrophy, six Limb-Girdle muscular dystrophies, and other CNS-related disorders. Sarepta is in a race to transform genetic knowledge into genetic medicine as every day is an opportunity to save lives from rare diseases.
This year’s TMP expands on the previously work completed by last year’s KGI team in support of Sarepta’s gene therapy team. Specifically, the objective of this year’s project was to define innovative technologies that would create a cohesive, easily accessible, and fluid patient experience before, during and after their treatment. This would enable Sarepta Therapeutics to provide a best-in-class patient treatment experience.

This year’s KGI team started with, and then significantly expanded the initial mobile app framework created last year. The project included generating a business plan for app development, identifying and rationalizing app functionalities, target app users, app data collection and analysis features, compliance considerations, and app development and deployment timelines. The team deliverables include suggestions for vendor partners, a budget analysis, and app prototypes that highlight key features and functionality. The team’s direction is based on insight gained from interviews with healthcare providers, caregivers, patients, and vendors.