Year: 2014-15
Company: Sarepta Therapeutics
Liaison(s): Bo Cumbo Emily Naughton
Sarepta Therapeutics is a leader in development of innovative RNA-based therapies for rare and orphan diseases. Originally founded as AVI Biopharma in 1980, Sarepta emerged as one of the frontrunners developing morpholino based antisense therapies for several indications. Sarepta’s clinical pipeline includes eteplirsen, an exon-skipping phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne’s Muscular Dystrophy (DMD), and other clinical candidates for the treatment of Ebola virus, Marburg virus, and Influenza. Sarepta is currently assessing new opportunities in rare and orphan disease. Previous TMP teams created a rare disease database, and made recommendations for areas to pursue based on assessments of feasibility and market potential. Sarepta is interested in building upon the work completed by previous teams in order to identify potential business opportunities. The 2014-2015 Sarepta TMP team initially conducted an exhaustive competitive analysis of RNA-based therapies in development in the US and EU. Based on diseases of interest identified during the initial phase of the project, the team conducted an intellectual property analysis to determine Sarepta’s freedom to operate in each space. The final phase of the project consisted of conducting market potential and feasibility analyses to pinpoint potential business opportunities for Sarepta. Completion of each element of this project has allowed provision of actionable recommendations regarding promising next steps for consideration.