Insights into causes of rare genetic immune disorders JExpMed
Disclosures: A. Lau reported being an employee of AstraZeneca since January 2023. J. Bier reported being an employee of Takeda Pharmaceutical since September 2022. R.S. Abraham reported personal fees from Enzyvant Therapeutics, Horizon Pharma, Beckman Coulter, and ClinGen outside the submitted work. C. Burkhart reported being an employee and shareholder of Novartis Pharma AG. Leniolisib is a p110δ-specific inhibitor and was discovered and developed by Novartis Pharma AG.
Leniolisib was provided in 2016 to S.G. Tangye for studies described in the manuscript. Leniolisib was outlicensed to Pharming Group N.V. in 2019. S.G. Tangye reported being on the Pharming Group NV Global Advisory Board for the use of leniolisib to treat individuals with inborn errors of immunity due to mutations in PIK3CD or PIK3R1. Pharming has licensed leniolisib from Novartis as a therapy for these conditions. E.K. Deenick reported “other” from CSL outside the submitted work.
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