Company intelligence / Profile preview

AntiVirals

Cambridge, Massachusetts, United States
Ownership
Private
Employees
~1300
Development stage
Other
01

Overview

Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on helping patients with rare, infectious, and other diseases. Originally founded as AntiVirals Inc. in 1980, the company pioneered the use of phosphorodiamidate morpholino oligomers (PMOs) for RNA-based therapeutics. Today, Sarepta is a leader in precision genetic medicine, with a multi-platform approach encompassing antisense oligonucleotides and gene therapy. The company has successfully brought several treatments for Duchenne muscular dystrophy (DMD) to market, including Exondys 51 and the first-ever gene therapy for DMD, Elevidys. Their extensive pipeline continues to target various forms of muscular dystrophy and other rare neuromuscular disorders.

Therapeutic areas
NeurologyRare Diseases
Modalities
Antisense oligonucleotideGene therapyRNA therapyPhosphorodiamidate morpholino oligomer (PMO)
Industry
Biotech
02

Partnerships

RocheDyno TherapeuticsSelecta BiosciencesGenEdit

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