Sarepta Therapeutics Inc.

Company Overview

Sarepta Therapeutics is a specialized biopharmaceutical company dedicated to develop RNA-based advanced therapies. The company is engaged in research and development activities for developing RNA for the treatment of neuromuscular diseases. It reported annual sales revenue of $84.4 million for the financial year 2018.

Sarepta Therapeutics’ vision is to change how the global methodologies the treatment of serious and dangerous diseases by opening the capability of RNA-based advances. The company’s main therapeutic areas include RNA-Targeted Therapies PMO, Gene Therapy and Gene Editing. The company’s top product is EXONDYS 51, intended to treat the patients of Duchenne muscular dystrophy (DMD). Another leading product called EXONDYS 51 is also intended to be used for the treatment of dystrophin disease in complex patients.

Sarepta Therapeutics’ product pipeline includes casimersen (SRP-4045), golodirsen (SRP-4053), Exon 52, SRP-5051, SRP-5045, SRP-5053, SRP-5052, SRP-5050, SRP-5044, GALGT2 (Nationwide), micro-dystrophin (Genethon), SRP-9001 micro-dystrophin (Nationwide), SRP-9003 (LGMD2E β-sarcoglycan), SRP-9005 (LGMD2C γ-sarcoglycan), SRP-9004 (LGMD2D α-sarcoglycan), MYO-201 (LGMD2B Dysferlin) ,CAPN-3 (LGMD2A Calpain 3), SRP-9006 (LGMD2L Anoctamin 5), CNS-1 (Lacerta), CNS-2 (Lacerta), SRP-9002 (MPS IIIA) (Lysogene) and neurotrophin 3 (CMT 1A).

Financial Highlights (FY 2024)

Company's Business Segments

• Product : EXONDYS 51, VYONDYS 53, AMONDYS 45, ELEVIDYS.
• Collaboration and other : Collaboration and other revenues primarily relate to collaboration arrangement with Roche.