Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics.
Our Portfolio
The following chart summarizes our main product candidate portfolio.
DEVELOPMENT PROGRAMS
ACTIVATED PI3Kδ SYNDROME (APDS)
Activated Phosphoinositide 3-kinase Delta (PI3Kδ) Syndrome, or APDS is a rare, genetic, and progressive primary immunodeficiency. Discovered in 2013, APDS is a disorder that impairs the immune system and the function of the white blood cells that recognize and attack viruses and bacteria to prevent infection.
PRIMARY IMMUNODEFICIENCIES (PIDs) WITH IMMUNE DYSREGULATION
Primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kẟ signaling in lymphocytes include ALPS-FAS, CTLA4 haploinsufficiency, NFKB1 haploinsufficiency and PTEN deficiency, among others. The unique genetic drivers in these PIDs lead to clinical phenotypes of immune dysregulation often shared with APDS.