Market Intelligence, Clinical Progress, and High-Purity Reagents for SCA2 & ALS Development.
TarMart Solution Ecosystem & Related Targets
Comprehensive reagent toolkit for ATXN2 drug discovery. Select your modality below:
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen (WT) | Human ATXN2 Full-Length Protein, WT (Q22). High purity (>95%), Endotoxin below 1 EU/µg. Sequence Verified. | View ATXN2 Products |
| Antigen (Mutant) | Human ATXN2 PolyQ-Expanded Mutant Proteins (Q58 / Q72 / Q104). Mimics pathogenic SCA2/ALS conformations. Aggregation competent. Theoretical MW confirmed. | View ATXN2 Products |
| Gene Delivery | ATXN2 Promise-ORF / Lentivirus. Full-length WT & Expanded alleles for stable neuronal cell line generation. | View ATXN2 Products |
| Knockdown Validator | ATXN2 siRNA Set (3 unique target sequences). For ASO/siRNA specificity and target engagement verification. | View ATXN2 Products |
| Benchmark Ab | Anti-ATXN2 Recombinant Antibody. Sequence-verified positive control for ICC/IHC and stress granule colocalization. | View ATXN2 Products |
| Related Target: ATXN1 | Ataxin-1 (SCA1). Parallel polyQ neurodegeneration pathway; essential for selectivity counter-screening. | View ATXN1 Products |
| Related Target: TARDBP | TDP-43 (TARDBP). ALS convergence node; stress granule dynamics and RNA metabolism overlap. | View TARDBP Products |
| Related Target: SOD1 | SOD1. Familial ALS target; combination therapy rationale with ATXN2 lowering strategies. | View SOD1 Products |
| Related Target: ATXN2L | Ataxin 2-Like Protein. Paralog with functional redundancy; critical for ASO counter-screening. | View ATXN2L Products |
| Related Target: PABPC1 | Poly(A)-Binding Protein Cytoplasmic 1. Key stress granule interactor; validates RNA-binding assays. | View PABPC1 Products |
| Related Target: C9orf72 | Primary genetic risk factor and biomarker in ALS/FTD. | View C9orf72 Products |
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| PolyQ length-dependent aggregation kinetics | WT (Q22) and pathogenic (Q58/Q72/Q104) ATXN2 proteins available; high purity (>95%) for ThT/SDD-AGE assays. Sequence Verified. |
| Intracellular knockdown specificity (ASO/siRNA) | Validated siRNA set included; sequence-confirmed for target engagement validation and rescue experiments. |
| Native RNA-binding & PPI conformation | Full-length, HEK293-expressed protein preserving native folding; Endotoxin Controlled. |
| Cross-species preclinical translation | Human/Mouse/Rat ortholog proteins with >95% purity; sequence verified by mass spectrometry. |
| Lack of Controls | Clinical Benchmark Antibodies (Biosimilars) and matched ORF Lentivirus included. |
| False Positives | Validated siRNA included for specificity checks in neuronal knockdown models. |
| Off-target ASO Toxicity | Validated siRNA included for specificity checks against ATXN2L. |
Live ATXN2 R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
Global Clinical Landscape & Future Outlook
The race for ATXN2 therapeutics is intensifying, with major players shifting focus from traditional small molecules to CNS-penetrant RNA-targeted modalities. As an intracellular RNA-binding protein, Ataxin-2 is a critical modifier of TDP-43 toxicity in Amyotrophic Lateral Sclerosis (ALS) and the direct causative agent of Spinocerebellar Ataxia type 2 (SCA2) via polyglutamine (polyQ) tract expansion. As first-generation ASO therapies advance toward clinical validation, the next wave of R&D is targeting precision allele-selective silencers and RNA-targeted small molecules that can modulate ATXN2 dosage without complete knockout.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| ASO / RNAi | Ionis, Biogen, Alnylam, Wave Life Sciences | SCA2, ALS, FTD | Knockdown efficiency assay (Need siRNA & qPCR-grade lysate controls) |
| Small Molecule (RNA modulators) | Expansion Therapeutics, academic consortia | SCA2 | RNA-binding competition assay (Need full-length native ATXN2 protein) |
| PROTAC / Degrader | Emerging biotech | ALS | Ternary complex formation (Need purified WT/Mutant ATXN2 + ligase) |
| Gene Therapy (AAV-shRNA) | Voyager, Novartis | SCA2 | Stable cell line generation (Need Lentivirus for neuronal transduction) |
Molecular Differentiation & Assay Strategy
PolyQ Length Specificity and Aggregation Kinetics
Pathogenic polyQ expansions (typically >Q34) lead to protein misfolding and stress granule pathology. Assays must distinguish between normal physiological (Q22) and pathological (Q58, Q72) aggregation propensities. TarMart provides strictly sequence-verified recombinant mutant proteins (Q22, Q58, Q72) with confirmed CAG repeat counts via mass spectrometry, endotoxin control (<1 EU/µg), and high purity (>95%) to avoid non-specific aggregation seeding.
Stress Granule Formation Capacity
ATXN2's pathological function is closely linked to stress granule dynamics. Drugs must be evaluated for their impact on SG formation or dissolution without affecting basal cellular functions. TarMart offers Lentivirus Premade Particles for stable expression of GFP-ATXN2(Q72) or mCherry-ATXN2 in neuronal cell lines (SH-SY5Y, PC12), ensuring proper post-translational modifications critical for SG localization.
Paralog Off-Target Risk Control (ATXN2L Counter-screening)
ATXN2L shares ~70% homology with ATXN2 in RNA-binding domains, with partial functional redundancy. ASOs or small molecules that also inhibit ATXN2L may cause unpredictable toxicity. TarMart provides ATXN2L recombinant protein as a counter-screening antigen and ATXN2 vs ATXN2L-specific siRNA sets for knockdown specificity validation.
Key Protein-Protein Interaction (PPI) Validation
ATXN2 interacts with PABPC1 and TDP-43 via its Lsm domain. Drugs may function by blocking these interactions. TarMart offers Co-IP validated anti-ATXN2 recombinant antibodies and PABPC1 recombinant protein as interaction controls for SPR or BLI experiments.