Pharming Group NV

Based in Leiden, the Netherlands, Pharming Group N.V. operates as a biopharmaceutical firm dedicated to advancing and marketing protein replacement therapies and precise medical solutions. Its global efforts, spanning the United States, Europe, and other international markets, focus on addressing rare diseases and critical unmet medical needs. The company's flagship product, Ruconest, is a recombinant human C1 esterase inhibitor prescribed for managing acute attacks of hereditary angioedema. Pharming's development pipeline also encompasses rhC1INH, which is under investigation for potential applications in pre-eclampsia, acute kidney injury, and COVID-19. Furthermore, they are progressing leniolisib, a phosphoinositide 3-kinase delta (PI3K delta) inhibitor, for individuals afflicted with activated PI3K delta syndrome, as well as an alpha-glucosidase therapy aimed at treating Pompe and Fabry diseases. The company maintains significant partnerships, including a development and license agreement with Novartis and a strategic alliance with Orchard Therapeutics plc. This latter collaboration involves the research, development, manufacture, and commercialization of OTL-105, an experimental ex-vivo autologous hematopoietic stem cell gene therapy designed to treat hereditary angioedema.