Sarepta Therapeutics Inc (Sarepta) discovers and develops novel therapeutics based on its RNA technology for the treatment of rare diseases including Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophies (LGMDs) and other neuromuscular and central nervous system (CNS) related disorders. PMO chemistry is a technology platform, which powers the design and development of novel treatments for rare, infectious, and other diseases. Its RNA technologies include phosphorodiamidate morpholino oligomers (PMOs) and peptide phosphorodiamidate morpholino oligomers (PPMOs). PMO chemistry forms the basis of its technology platform, which powers the design and development of novel treatments for rare, infectious and other diseases.

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The R&D of Sarepta focuses on the development of innovative products and discovering, developing, manufacturing, and delivering therapies to patients with rare diseases. In FY2022, Globus spent US$877.09 million on its R&D activities, amounting to 13.7% of the company's total revenue. The company holds numerous issued patents and exclusive rights to issued patents. It has 779 employees that are engaged directly in research and development activities. It also owns and has licenses to various patent applications, in each case in the US and other countries.