Sarepta Therapeutics, Inc. (NASDAQ: SRPT)

Sarepta Therapeutics, Inc. (SRPT) is a company that operates in the biotechnology industry, focusing on the development and commercialization of RNA-targeted therapeutics and gene therapy products for the treatment of rare diseases.

The company's main business activities include the development of proprietary RNA-targeted therapeutics and gene therapy products for the treatment of rare diseases. Sarepta's operations span across various countries, with a focus on Duchenne muscular dystrophy (Duchenne) and Limb-girdle muscular dystrophies (LGMDs).

Sarepta generates revenue primarily through the sale of its approved products, including EXONDYS 51, VYONDYS 53, AMONDYS 45, and ELEVIDYS. EXONDYS 51 is a RNA-targeted therapeutic that treats Duchenne in patients with a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping. VYONDYS 53 and AMONDYS 45 are also RNA-targeted therapeutics that treat Duchenne in patients with a confirmed mutation of the dystrophin gene that is amenable to exon 53 or 45 skipping, respectively. ELEVIDYS is a gene therapy product that delivers a functional copy of the dystrophin gene to the muscles of patients with Duchenne.

Sarepta holds a strong position within the industry, with key patents and regulatory exclusivities protecting its products and product candidates. EXONDYS 51, for instance, has been granted data exclusivity until June 22, 2035, and Orphan Drug Exclusivity until June 22, 2030. VYONDYS 53 has been granted new chemical entity ("NCE") exclusivity until December 12, 2024, and Orphan Drug Exclusivity until December 12, 2026. AMONDYS 45 has been granted NCE exclusivity until February 25, 2026, and Orphan Drug Exclusivity until February 25, 2028.

Sarepta's products are designed to treat Duchenne by skipping specific exons of the dystrophin gene, allowing for the production of a functional dystrophin protein. The company's gene therapy products, including ELEVIDYS and SRP-9003, use AAV vectors to deliver functional copies of specific genes to the muscles of patients with rare genetic disorders.

In terms of its competitive position, Sarepta competes with other companies in the biotechnology industry that are developing and commercializing RNA-targeted therapeutics and gene therapy products for the treatment of rare diseases. Some of the company's competitors include Biogen, Inc., Pfizer, Inc., and Novartis AG.

Sarepta's customer base includes hospitals, clinics, and pharmacies that purchase its products. The company's products are used to treat patients with rare genetic disorders, and are typically prescribed by physicians and other healthcare professionals.

Sarepta's brand names and trade names include EXONDYS 51, VYONDYS 53, AMONDYS 45, ELEVIDYS, and SRP-9003. These products are crucial to the company's operations and revenue generation.