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A biotechnology company focused on developing transformative medicines for serious diseases with a specialty in cystic fibrosis and genetic disorders.
The company is a global biotechnology firm predominantly engaged in the development and commercialization of transformative therapies for serious diseases, with a primary focus on specialty markets. It has developed four approved medications for cystic fibrosis (CF) and one for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This innovative firm is also advancing a diverse pipeline, including therapies for various conditions such as acute and neuropathic pain, APOL1-mediated kidney disease, type 1 diabetes, and myotonic dystrophy type 1, aiming to address the underlying causes of these diseases and improve patient outcomes.
With a core strategy centered on scientific innovation and serial innovation, the company aims to expand treatment options and market reach for existing medications while also developing novel therapies that target causal biology. The company's business model combines internal research, collaborations, and acquisitions to sustainably enhance its R&D capabilities, ensuring a robust pipeline and continued leadership in the specialty biopharmaceutical sector across multiple disease areas.
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