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BridgeBio Pharma is a biotechnology company focused on discovering, developing, testing, and delivering transformative treatments for patients with genetic diseases. The company has four programs in its late-stage pipeline focusing on Mendelian disorders, oncology, and gene therapy. One of its key programs, acoramidis (AG10), is an orally administered small molecule designed to stabilize tetrameric transthyretin for the treatment of transthyretin amyloid cardiomyopathy.
Ultragenyx Pharmaceutical Inc is a USA-based biopharmaceutical company. It identifies, acquires, develops, and commercializes novel products for the treatment of serious rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. The company's medicine portfolio includes Crysvita, Dojolvi, and Mepsevii. Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 1 year of age and older. Mepsevii is indicated in pediatric and adult patients for the treatment of Mucopolysaccharidosis VII.