Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Elongation factor G 1, mitochondrial (GFM1)
- Target
- GFM1
- Molecular classification
- Enzyme (specifically a GTPase, mitochondrial translation elongation factor), Other (nuclear-encoded mitochondrial protein involved in translation)
01
Overview
Elongation factor G 1, mitochondrial (GFM1) is a nuclear-encoded mitochondrial translation elongation factor essential for the synthesis of proteins from mitochondrial DNA. It acts as a GTPase to promote the translocation of tRNAs and movement along the mitochondrial ribosome during peptide elongation. This function is critical for the assembly of mitochondrial respiratory chain complexes required for energy production (oxidative phosphorylation). Genetic defects in GFM1 lead to combined oxidative phosphorylation deficiency 1, manifesting as severe neurological and hepatic disease, and sometimes Leigh syndrome, with high lethality in childhood. Currently, GFM1 is not a direct target of any therapeutic drugs.
- Other names
- G elongation factor mitochondrial 1EFGEFG1GFMEF-GmtmEF-G 1hEFG1EFGMEGF1mtEF-G1COXPD1G translation elongation factor, mitochondrialMitochondrial elongation factor GMitochondrial elongation factor G1
02
Mechanism of action
None established. There are no drugs acting on GFM1 at present.
03
Biological functions
Mitochondrial translational elongation: Facilitates protein synthesis within mitochondria by promoting the translocation step during translationGTPase activity: Hydrolyzes GTP as part of its function in coordinating tRNA and ribosome movementEnergy metabolism: Indirectly essential for oxidative phosphorylation by enabling synthesis of mitochondrially encoded OXPHOS subunits
04
Disease associations
Combined oxidative phosphorylation deficiency 1: Severe early-onset metabolic disorder affecting neurological and hepatic function, due to impaired mitochondrial translationMitochondrial disease with hepatopathy: Liver involvement, lactic acidosis, developmental delayLeigh syndrome (in some cases)
05
Safety considerations
Essentiality for mitochondrial function: Complete loss of function is incompatible with life
06
Biomarkers
GFM1 gene variants: Pathogenic loss-of-function alleles are molecular biomarkers for diagnosing combined oxidative phosphorylation deficiency 1
Beyond the preview
Go deeper on Elongation factor G 1, mitochondrial (GFM1).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Clinical trials
Full profile accessFollow the clinical studies evaluating therapies directed at this target.
- Trial design
- Status
- Readouts
Competitive landscape
Full profile accessCompare approaches across drug candidates, modalities, and indications.
- Programs
- Modalities
- Indications
Literature & evidence
Full profile accessInvestigate the research and source evidence behind target biology and development.
- Publications
- Sources
- Analysis
Patents
Full profile accessExplore patent activity around therapies and technologies addressing this target.
- Patents
- Assignees
- Technologies
Research & analysis
Full profile accessConnect target biology, drug development, and emerging evidence in your research.
- Biology
- Development news
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on Elongation factor G 1, mitochondrial (GFM1).