Company: Sarepta Therapeutics
Liaison(s): Bo Cumbo
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.