Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Survival Motor Neuron 1
- Molecular classification
- RNA-binding protein, Transcription factor, Other
01
Overview
Survival motor neuron 1 (SMN1) is a gene that encodes the survival motor neuron (SMN) protein, which is essential for the maintenance and function of specialized nerve cells called motor neurons. The SMN protein is ubiquitously expressed throughout the body, with its highest concentrations in the spinal cord. The SMN protein forms part of a larger complex known as the SMN complex and plays a critical role in maintenance and survival of motor neurons, assembly and biogenesis of small nuclear ribonucleoproteins (snRNPs), and processing messenger RNA (mRNA). Mutations or deletions in *SMN1* cause spinal muscular atrophy (SMA).
- Other names
- SMN1Survival motor neuron protein
02
Mechanism of action
Enhancement of SMN protein levels via splicing modulation or other mechanisms.
03
Biological functions
snRNP assemblymRNA splicingmRNA processingmRNA traffickingNeuronal developmentMaintenance of motor neuronsRegulation of transcriptionTelomerase regenerationCellular traffickingDNA repairTranslationSelenoprotein synthesisMacromolecular traffickingStress granule formationCell signalingCytoskeleton maintenance
04
Disease associations
Spinal muscular atrophy (SMA)Male infertilityInclusion body myositisAmyotrophic lateral sclerosis (ALS)Osteoarthritis
05
Safety considerations
Potential for off-target effects due to widespread expression and multiple functions of SMN protein.Challenges in achieving optimal SMN protein levels in all affected tissues.
06
Biomarkers
SMN protein levels in blood or tissues
Beyond the preview
Go deeper on Survival Motor Neuron 1.
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 Survival Motor Neuron 1.