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Drug Profile

Research programme: exon targets therapeutics - Sarepta Therapeutics

Latest Information Update: 30 Jun 2023

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At a glance

Originator Sarepta Therapeutics
Class Antisense oligonucleotides; Morpholines; Oligonucleotides; Peptide drug conjugates; Peptides
Mechanism of Action Dystrophin expression modulators

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Preclinical Duchenne muscular dystrophy

Most Recent Events

15 Jun 2023 Preclinical trials in Duchenne muscular dystrophy in USA (Parenteral) (Sarepta Therapeutics pipeline, June 2023)

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