Market Intelligence, Clinical Progress, and High-Purity Reagents for Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) Development.
TarMart Solution Ecosystem & Related Targets
Comprehensive reagent toolkit for UNC13A drug discovery. Select your modality below:
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen | UNC13A Recombinant Protein (Wild-Type & ALS-Risk Domain Variants) HEK293 Expressed. High purity (>95%), Endotoxin <1 EU/µg. Sequence Verified. Theoretical MW confirmed. |
View UNC13A Products |
| Gene Delivery | UNC13A Promise-ORF / Lentivirus Premade Particles Full-length ORF for stable neuronal cell line construction. Endotoxin Controlled. |
View UNC13A Products |
| Benchmark Ab | Anti-UNC13A Recombinant Antibody (Reference Sequence) Positive control for IP, WB, and target engagement. Sequence Verified. |
View UNC13A Products |
| Validator | UNC13A siRNA Set (3 unique sequences) For knockdown and specificity verification. |
View UNC13A Products |
| Related Target A | TARDBP (TDP-43) Upstream RNA-binding protein regulating UNC13A cryptic splicing. |
View TARDBP Products |
| Related Target B | STMN2 Parallel cryptic exon target in ALS/FTD; co-pathway readout. |
View STMN2 Products |
| Related Target C | UNC13B Paralog family member for selectivity and off-target profiling. |
View UNC13B Products |
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| Cryptic Exon Splicing Detection | Full-length ORF Lentivirus maintaining native splice site architecture; Sequence Verified |
| C2 Domain Lipid Binding Validation | High-purity C2 domain fragments (>95%) with endotoxin control for DAG/phospholipid SPR |
| Cross-species Translation (Human/Mouse/Cyno) | Human, Mouse, and Cynomolgus UNC13A ortholog proteins available (>95% purity, sequence verified) |
| Paralog Selectivity (UNC13B/UNC13C) | Homolog panel proteins strictly verified by mass spec; domain boundary optimized |
| Lack of Qualified Immunoreagents | Recombinant anti-UNC13A antibodies with defined sequences; Endotoxin-controlled (<1 EU/µg) |
| Off-Target Knockdown Artifacts | Validated siRNA set included for rescue and specificity checks |
Live UNC13A R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
Global Clinical Landscape & Future Outlook
UNC13A has emerged as a critical downstream effector of TDP-43 proteinopathy in Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). Aberrant splicing of UNC13A cryptic exon occurs in up to 97% of ALS cases and 50% of FTD cases. The main therapeutic strategies include antisense oligonucleotides (ASOs) targeting the cryptic splice site (e.g., Biogen, Ionis, QurAlis), small molecule stabilizers of the C2 domain (academic consortia, Stealth Biotech), and gene therapy (4D Molecular Therapeutics, various biotechs). As first-generation therapies reach the clinic, the next wave focuses on sequence-specific steric blocking and combination therapies addressing TDP-43 proteinopathy.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| ASO (Antisense Oligonucleotide) | Biogen, Ionis, QurAlis | ALS, FTD | Splicing reporter stable cell lines (Lentivirus-based); isoform-specific antibody detection |
| Small Molecule (C2 Stabilizer) | Academic Consortia, Stealth Biotech | Sporadic ALS | Lipid binding displacement assay (Need high-purity C2 domain protein) |
| Gene Therapy (AAV) | 4D Molecular Therapeutics, Various Biotechs | ALS | Functional rescue in neuronal models (Need full-length ORF Lentivirus) |
Additional Key Functional Domains & Mutations
UNC13A contains three functional domains: C2 1, C2 2, and MHD1. Key mutations linked to NEDHES (Neurodevelopmental Disorder with Epilepsy and Hypomyelination, Spasticity, and Cerebellar Ataxia) have been identified: likely pathogenic loss-of-function variants (VAR_091494, VAR_091495) and a variant of uncertain significance (VAR_091496). These mutations impair synaptic function and are relevant to disease models.