Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants
Beth Israel Deaconess Medical Center · Georgetown University · +12 more institutions
Abstract
Persons with toxic gain-of-function variants in the gene encoding apolipoprotein L1 (APOL1) are at greater risk for the development of rapidly progressive, proteinuric nephropathy. Despite the known genetic cause, therapies targeting proteinuric kidney disease in persons with two APOL1 variants (G1 or G2) are lacking. Download a PDF of the Research Summary. We used tetracycline-inducible APOL1 human embryonic kidney (HEK293) cells to assess the ability of a small-molecule compound, inaxaplin, to inhibit APOL1 channel function. An APOL1 G2–homologous transgenic mouse model of proteinuric kidney disease was used to assess inaxaplin treatment for proteinuria. We then conducted a single-group, open-label, phase 2a…
Citation impact
- FWCI
- 34.79
- Percentile
- 100%
- References
- 38
Authors
19- OEOgo EgbunaCorresponding
Beth Israel Deaconess Medical Center, Georgetown University, Université Paris Cité, Vertex Pharmaceuticals (United States)
- BZBrandon Zimmerman
Beth Israel Deaconess Medical Center, Georgetown University, Université Paris Cité, Vertex Pharmaceuticals (United States)
- GMGeorge Manos
Beth Israel Deaconess Medical Center, Georgetown University, Université Paris Cité, Vertex Pharmaceuticals (United States)
- AHA H Fortier
Beth Israel Deaconess Medical Center, Georgetown University, Université Paris Cité, Vertex Pharmaceuticals (United States)
- MCMadalina Chirieac
Beth Israel Deaconess Medical Center, Georgetown University, Université Paris Cité, Vertex Pharmaceuticals (United States)
Topics & keywords
- Disease
- Kidney disease
- Kidney
- Genetics
- Medicine
- Biology
- Evolutionary biology
- Internal medicine
- Good health and well-being