Liaison(s): Allicyn Aubut, Tino Quintero, Emily Regan
Sarepta is leading a revolution in precision genetic medicine—every day is an opportunity to change the lives of people living with rare disease. The company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis Type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy, and gene editing.
Sarepta is developing a commercial site readiness strategy for the potential launch of their in-vivo micro-dystrophin gene therapy product for DMD (SRP-9001). The product is a novel therapy for the DMD population and will disrupt current treatment pathways; clinical protocols and institutional processes will need to change so institutions can provide this therapy. In order to ensure high- quality support and a smooth launch, the Sarepta Team Master’s Project (TMP) team was tasked with determining a “super analog” for Sarepta to consider as they evolve their site readiness strategy. This involved performing analysis on innovative treatments, disruptive products, one-time procedures, digital applications, and centers of excellence. In identifying and researching different models, the TMP team summarized common pain points, best practices, and innovative solutions to provide suggestions for all steps in the patient journey, taking into consideration multiple participants involved in gene therapy administration (patient, caregiver, provider, and institution). Completion of this project has provided Sarepta with important information to holistically support the DMD community and solve challenges of gene therapy commercialization.