Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
ORMDL sphingolipid biosynthesis regulator 1 (ORMDL1)
- Target
- ORMDL1
- Molecular classification
- Regulatory protein, Endoplasmic reticulum transmembrane protein, Enzyme regulator (negative regulator of serine palmitoyltransferase activity)
01
Overview
ORMDL sphingolipid biosynthesis regulator 1 (ORMDL1) is a small transmembrane regulatory protein located in the endoplasmic reticulum, highly conserved across species and part of the ORMDL family (ORMDL1, ORMDL2, ORMDL3)[1][2][4][5]. ORMDL1 is a negative regulator of serine palmitoyltransferase (SPT), the enzyme catalyzing the first committed step in sphingolipid biosynthesis. By sensing elevated ceramide levels, ORMDL1 inhibits SPT activity, acting as a key homeostatic checkpoint to prevent toxic ceramide accumulation and maintain sphingolipid balance. In mammals, loss or dysregulation of ORMDL1 has been linked to abnormal myelination, cancer progression, and modulation of inflammatory and neurodegenerative processes. ORMDL1 function as a therapeutic target is under investigation, particularly in oncology and neurodegeneration, but no direct small-molecule drugs or antagonists are currently established[1][2][4][5].
- Other names
- ORMDL1ORML1adoplin-1
02
Mechanism of action
Inhibition of serine palmitoyltransferase via feedback from ceramide binding; Modulation of sphingolipid metabolic pathway enzymes
03
Biological functions
Regulation of sphingolipid biosynthesisNegative regulation of ceramide biosynthetic processLipid homeostasisPotential modulation of gamma-secretase activity
04
Disease associations
Neurodegenerative disease (e.g., involvement in amyloid precursor protein processing relevant to Alzheimer’s disease)Inflammation (regulation of sterile inflammation via ceramide control)Cancer (overexpressed in certain cancers and linked to prognosis, e.g., diffuse large B cell lymphoma)Neuropathy (Hereditary Sensory And Autonomic Neuropathy Type 1)Retinitis Pigmentosa 26
05
Safety considerations
Potential for disruption of sphingolipid homeostasis leading to myelination defects when dysregulatedPossible impact on programmed cell death (apoptosis) if ceramide accumulates excessively
06
Biomarkers
Prognostic biomarker in some cancers (e.g., diffuse large B cell lymphoma)
Beyond the preview
Go deeper on ORMDL sphingolipid biosynthesis regulator 1 (ORMDL1).
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 ORMDL sphingolipid biosynthesis regulator 1 (ORMDL1).