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RLT-72484
- Development stage
- Preclinical
- Lead developer
- Ridgeline Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
RLT-72484 is an orally bioavailable small molecule inhibitor of Nicotinamide N-methyltransferase (NNMT) being developed by Ridgeline Therapeutics for the treatment of Duchenne Muscular Dystrophy (DMD). The drug targets NNMT, an enzyme that plays a key role in muscle metabolism and stem cell health. By inhibiting NNMT, RLT-72484 aims to reactivate dysfunctional and senescent muscle stem cells (muSC), improve muscle mitochondrial function, and enhance overall muscle strength and regeneration. Originally identified by researchers at the University of Texas Medical Branch, the program has demonstrated the ability to restore muscle health in animal models of muscular dystrophy and is currently in the preclinical development stage.
- Other names
- NNMT inhibitor for DMDNNMT inhibitor (Duchenne Muscular Dystrophy program)
02
Targets
NNMT (Nicotinamide N-methyltransferase)
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