Pharming Group N.V. (PHGUF) Research

Pharming Group N.V. operates as a biopharmaceutical firm focused on creating and commercializing protein replacement therapies and precision medicines. These specialized treatments target rare diseases and areas with critical unmet medical needs, reaching patients throughout the United States, Europe, and other international regions. Pharming's flagship therapeutic is Ruconest, a genetically engineered human C1 esterase inhibitor designed to manage acute episodes of hereditary angioedema. Beyond its primary offering, the company's pipeline actively explores the use of rhC1INH for conditions such as pre-eclampsia, acute kidney injury, and COVID-19. Additionally, Pharming is developing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor, for individuals suffering from activated PI3K delta syndrome, alongside an alpha-glucosidase therapy addressing Pompe and Fabry diseases. The company maintains a development and licensing collaboration with Novartis. Moreover, a strategic alliance with Orchard Therapeutics plc involves the research, development, manufacturing, and commercialization of OTL-105. This investigational ex-vivo autologous hematopoietic stem cell gene therapy aims to treat hereditary angioedema. Its corporate base is located in Leiden, the Netherlands.

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