- In April 2025, researchers at the Gladstone Institutes in the U.S. reported promising results from a study involving a repurposed compound named HypoxyStat, which mimics the physiological effects of low-oxygen environments. Used in mice with Leigh syndrome, the drug extended lifespan by more than threefold and significantly improved neurological symptoms. This breakthrough points to a new therapeutic avenue for mitochondrial diseases and underscores the potential of hypoxia-mimicking agents in drug repurposing strategies
- In March 2025, Japanese scientists developed an innovative enzyme-based gene-editing system (mpTALENs) to correct mutations in mitochondrial DNA using patient-derived stem cells. This technology offers precision correction and presents a new frontier for mitochondrial drug repurposing and therapeutic interventions, especially for conditions like MELAS and MERRF syndromes
- In February 2025, NeuroVive Pharmaceutical advanced the development of KL1333, a repurposed NAD+ modulator, into early-phase clinical trials. The drug demonstrated improvements in muscle endurance and reduced fatigue in patients with mitochondrial myopathy, reinforcing its potential as a key treatment for multiple mitochondrial disorders
- In January 2025, the United Mitochondrial Disease Foundation (UMDF), in collaboration with Mayo Clinic, launched a screening project evaluating hundreds of approved compounds in cardiac cell models derived from MELAS patients. The aim is to identify off-label drugs that can reverse mitochondrial dysfunction, with selected candidates advancing toward clinical trials
