Sarepta Therapeutics

Sarepta Therapeutics is an American biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics, gene therapy, and other genetic medicine approaches for the treatment of rare diseases. The company is primarily known for its work in Duchenne muscular dystrophy (DMD) treatments.

History

Founded in 1980 as AntiVirals Inc., the company initially focused on developing treatments for infectious diseases. In 2003, it was renamed AVI BioPharma and shifted its focus to RNA-based therapeutics. In 2012, the company adopted its current name, Sarepta Therapeutics, and began to concentrate on rare genetic disorders, particularly DMD.

Research and Development

Sarepta Therapeutics' research and development efforts are primarily focused on genetic diseases, with a significant emphasis on DMD. The company's lead product, Eteplirsen (brand name Exondys 51), received accelerated approval from the U.S. Food and Drug Administration (FDA) in 2016. This marked the first FDA-approved treatment specifically for DMD.

The company is also developing other RNA-targeted and gene therapy candidates for the treatment of DMD and other rare genetic diseases. These include Golodirsen (Vyondys 53) and Casimersen (Amondys 45), both of which have received FDA approval.

Financials

As a biopharmaceutical company, Sarepta Therapeutics' revenue primarily comes from the sales of its approved drugs. The company also receives funding from various research grants and collaborations with other pharmaceutical and biotechnology companies.

Criticism and Controversy

Sarepta Therapeutics has faced criticism and controversy, particularly regarding the accelerated approval of Eteplirsen by the FDA. Critics argue that the approval was based on insufficient evidence of the drug's effectiveness.

See Also

• Biopharmaceutical
• Gene therapy
• Duchenne muscular dystrophy
• Eteplirsen
• Golodirsen
• Casimersen