Year: 2018-19

Company: Sarepta Therapeutics, Inc.

Liaison(s): Damon Asher, Pratik Parikh, Emily Regan

Sarepta Therapeutics, Inc. is at the forefront of precision genetic medicine, having built a world-leading pipeline for the treatment of Duchenne muscular dystrophy (DMD) across several therapeutic modalities, including RNA, gene therapy and gene editing. The company has recently expanded its portfolio to include Limb-girdle muscular
dystrophy (LGMD), Pompe, Charcot Marie Tooth (CMT) Disease, Mucopolysaccharide 3A disease, and central nervous system (CNS) disorders to further increase the impact of their therapies on patients with rare and neuromuscular diseases. Sarepta’s mission is to forever change the course of rare and neuromuscular diseases so patients can dream big, live fully, and journey far.

With the recent rapid expansion of gene therapy treatment and disease areas within Sarepta, there is a need to increase internal expertise in these new areas and prepare for potential launches. The Sarepta Team Master’s Project team was tasked with creating educational materials and guides to help on-board new employees on the basics of gene therapy. Additionally, the TMP team is performing deep dive analyses into four disease groups, including LGMD, CMT 1A, Pompe, and MPSIIIA, especially in relation to the gene therapy landscape and potential to treat these four disease areas. Focus areas will include pathophysiology, epidemiology, natural history/disease state, and an investigation of all pre-clinical and clinical work on therapeutic approaches. The project will culminate with a 5-year look-ahead and recommendation from the KGI TMP team on what the future therapeutic landscape may look like for each of those specific disease states.