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R007
- Development stage
- Preclinical
- Lead developer
- RiboNova
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
R007 is a small molecule drug, with probucol as its active ingredient, developed by RiboNova for the treatment of all forms of genetically-confirmed mitochondrial disease. It functions by targeting the nutrient-sensing signaling network in the cytosol to reverse the dysregulation of key signaling nodes. Specifically, R007 acts as an inhibitor of AMP-activated protein kinase (AMPK) and mammalian target of rapamycin complex 1 (mTORC1), and as an antagonist of peroxisome proliferator-activated receptor (PPAR). While a tablet formulation is being evaluated in Phase 2 for adults, an oral liquid formulation is currently in preclinical development (formulation stage) specifically for pediatric use in infants and children.
- Other names
- probucol
02
Targets
Mechanistic target of rapamycin complex 1AMPK (Adenosine monophosphate–activated protein kinase)
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