Examples of approved drugs with causal support from tissue-specific human autoimmunity or human genetics. SOST, sclerostin; IL6R, interleukin 6 receptor; PCSK9, proprotein convertase subtilisin/kexin type 9; GLP1R, glucagon-like peptide–1 receptor; IL23A, interleukin 23α; IL12B, interleukin 12β.
Clinical disorder | Tissue-specific autoimmunity | Drug |
Encephalitis | DRD2 | Antipsychotics |
Narcolepsy | Orexin neurons | Suvorexant |
Diabetes | Pancreatic beta cells | Insulin |
Myasthenia gravis | Acetylcholine receptors | Acetylcholinesterase inhibitors |
Thrombotic thrombocytopenic purpura | ADAMTS13 on platelets | Caplacizumab* |
Clinical disorder | Gene(s) | Drug(s) |
Schizophrenia | DRD2 | Antipsychotics |
Sclerosteosis and van Buchem disease | SOST | Romosozumab* |
Rheumatoid arthritis | IL6R | Tocilizumab |
High cholesterol | PCSK9 | Evolocumab and alirocumab |
Diabetes | GLP1R | Incretin mimetics |
Psoriasis | IL23A, IL23R, and IL12B | Ustekinumab |
Atopic dermatitis | IL4RA | Dupilumab* |
*Not yet approved.