Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Mitochondrial calcium uniporter (MCU) is a highly selective calcium-conducting channel located in the inner mitochondrial membrane, responsible for the primary pathway of calcium entry into the mitochondrial matrix (UniProt Q9BPX6). It functions as part of a large protein complex that includes regulatory subunits such as MICU1, MICU2, and EMRE to maintain calcium homeostasis (PMID: 21666605). By regulating mitochondrial calcium levels, MCU plays a critical role in modulating aerobic metabolism, ATP production, and cell death pathways such as apoptosis and necrosis (PMID: 30104731). Dysregulation of MCU-mediated calcium uptake is implicated in various pathologies, including myocardial infarction, where calcium overload leads to tissue damage, and neurodegenerative diseases like Alzheimer's and Parkinson's (PMID: 31434801). In cancer, MCU expression often correlates with tumor progression and chemoresistance by influencing metabolic reprogramming and survival signaling (PMID: 30610213). While several small molecules like Ru360 and DS16570511 have been developed to inhibit MCU activity in research settings, therapeutic targeting remains challenging due to the channel's essential role in systemic energy homeostasis (PMID: 32561583).
Inhibition or activation of mitochondrial calcium uptake through the inner mitochondrial membrane.
4 more in the full profile.
Beyond the preview
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Explore the programs pursuing this target and their development progress.
Follow the clinical studies evaluating therapies directed at this target.
Compare approaches across drug candidates, modalities, and indications.
Investigate the research and source evidence behind target biology and development.
Explore patent activity around therapies and technologies addressing this target.
Connect target biology, drug development, and emerging evidence in your research.
See how Gosset can support your research on Mitochondrial calcium uniporter (MCU) (MCU).