Can you explain what REN001 that is being developed by Reneo Pharmaceuticals is supposed to do? Is it related to the PPARS at all or is it a completely different drug? Thank you so much!
Stephanie, REN-001 is being developed by Reneo Pharmaceuticals and is a PPAR delta agonist. All of all the genes that make fatty acid proteins, and many related to other energy producing cellular functions (the respiratory chain) are contained in the cell nucleus. They are controlled by a common genetic sequence known as a promotor, in this case the PPAR delta promotor. REN-001 binds this promotor and increases the productions of all of the genes that it controls. There are several potential benefits to this increase. First, if a patient has a mutation in an FAO gene that leaves a protein with a little bit of activity, increasing the amount of enzyme increase total cellular activity by a little bit. Second, increasing the other energy pathways may improve overall energy generation in cells even when FAO is not functioning properly. Finally, the PPAR delta promotor also controls genes involved in decreasing cellular inflammation that in turn helps reduce the accumulation of a damaging compound in the cell known as reactive oxygen species. In cellular studies, my lab has shown that the combined effect of treating cells from patients with long chain FAODs with this drug is to improve cellular energy production. A phase one (safety) trial is currently in progress to test REN-001 in patients with long chain FAODs. For now the study is limited to patients older than age 18, but it will move to younger children as we demonstrate its safety in the older patients. Anyone interested in participating in the study can get more information on clinicaltrials.gov or by contacting me directly. Hope this helps. Dr. Vockley
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