Sarepta Therapeutics

US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) – formerly AVI BioPharma is focused on developing first-in-class, RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases.

Sarepta'’s unique technology has yielded a diverse pipeline of RNA-based therapeutics that include its lead clinical candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy, a debilitating and life-threatening genetic disorder, as well as potential treatments for some of the world’'s most lethal infectious diseases.

Its core phosphorodiamidate morpholino oligomer (PMO) antisense technology is uniquely versatile, allowing for the development of a range of therapeutic candidates that target different types of RNA.

Unlike most other RNA-based approaches, such as small interfering RNA (siRNA), Sarepta’'s technology can directly target both messenger RNA (mRNA) and precursor messenger RNA (pre-mRNA) to either down-regulate or up-regulate targeted genes or proteins (i.e. turn “on” or turn “off” production of a target protein).

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