Sarepta Therapeutics

[5][6] In July 2009, the company announced they would move their headquarters from Portland, Oregon, north to Bothell, Washington, near Seattle.

[11] In June 2017, Sarepta Therapeutics appointed former Allergan executive Doug Ingram as its new CEO to replace Edward Kaye who had announced plans to step down earlier that year.

[12] In February 2019, Sarepta acquired five gene therapy candidates for $165 million after one of them, MYO-101, produced results with a new gene therapy candidate for patients with Limb-Girdle muscular dystrophy; two months after receiving a single treatment, muscles from all three patients were producing the protein they couldn't make on their own.

Morpholinos can also work as splice-switching oligos, targeting pre-mRNA to alter splicing and so causing changes in the structure of the mature mRNA (the mechanism of the approved drug eteplirsen).

In March 2013, the Company announced positive results from a non-human primate study of AVI-7288, the drug candidate for treatment of Marburg virus infection.

[22] In December 2019, golodirsen (Vyondys 53) received US FDA approval[23] for the treatment of cases that can benefit from skipping exon 53 of the dystrophin transcript.

[25] In June 2023, ELEVIDYS (Delandistrogene moxeparvovec) was approved by the FDA for use in 4-5 year old boys with mutations in the DMD gene.

[28][29] In June 2024, the U.S. Food and Drug Administration expanded approval of delandistrogene moxeparvovec to include individuals with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene who are at least 4 years of age.