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Profile
From SEC filingsCompany description
We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying our proprietary, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne muscular dystrophy ("Duchenne") and are developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne and LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead for the treatment of Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 (“DM1”), Spinocerebellar ataxia ("SCA"), Idiopathic Pulmonary Fibrosis ("IPF"), Huntington's disease and other neuromuscular and skeletal diseases. To date, we have developed and commercialized the following four approved products for the treatment of Duchenne: EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”), AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), and ELEVIDYS. Each of these approved products, and the indications for which they have been approved for, is described under the heading “Our Commercial Products” in this Item 1.
Read the full filing description · filed 2 Mar 2026
At a glance
- Sector
- Healthcare
- Industry
- Pharmaceutical Preparations
- CEO
- Douglas S. Ingram
- Employees (2026)
Source: SEC filings. Information and analytics, not investment advice.