Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Serine palmitoyltransferase long chain base subunit 1 (SPTLC1)
- Target
- SPTLC1
- Molecular classification
- Enzyme, Long Chain Base Subunit
01
Overview
Serine palmitoyltransferase long chain base subunit 1 (SPTLC1) is a key enzyme involved in the de novo synthesis of sphingolipids. It forms part of the serine palmitoyltransferase (SPT) complex, which catalyzes the initial and rate-limiting step in this pathway: the condensation of L-serine and palmitoyl-CoA. Sphingolipids are critical components of cell membranes and play vital roles in various cellular processes, including cell signaling and membrane structure. Mutations in SPTLC1 are associated with hereditary sensory neuropathy type IA (HSN I), a disorder characterized by peripheral nerve degeneration. Some mutations can result in the production of atypical and toxic deoxysphingoid bases.
- Other names
- HSAN1HSN1LBC1LCB1SPTI
02
Mechanism of action
Inhibition of sphingolipid biosynthesis
03
Biological functions
Sphingolipid metabolismMembrane integrityCell-cell communicationSignal transduction pathwaysRegulation of apoptosisRegulation of proliferationVascular developmentEndothelial cell function
04
Disease associations
Hereditary sensory neuropathy type IAMacular diseaseJuvenile amyotrophic lateral sclerosis
05
Safety considerations
Potential neurotoxicity due to accumulation of deoxysphingoid bases with certain mutations.Off-target effects on other metabolic pathways.
Beyond the preview
Go deeper on Serine palmitoyltransferase long chain base subunit 1 (SPTLC1).
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 Serine palmitoyltransferase long chain base subunit 1 (SPTLC1).