TarMart Solution Ecosystem & Related Targets
Comprehensive reagent toolkit for Huntingtin drug discovery. Select your modality below:
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen (Wild-Type) | HTT N-terminal (1-588) WT Control (Q23); also Exon-1 WT 23Q available. High purity (>95%), Endotoxin <1EU/µg. Sequence Verified. | View HTT Products |
| Antigen (Mutant) | HTT N-terminal (1-588) PolyQ Mutant variants (Q73, Q103); also Exon-1 Mutant 73Q. High purity (>95%), Endotoxin <1EU/µg. Sequence Verified by MS. | View HTT Products |
| Gene Delivery | HTT Promise-ORF / Lentivirus particles for full-length ORF (WT or Mutant exon-1). Ready-to-transduce for stable cell lines. | View HTT Products |
| Benchmark Ab | Anti-HTT recombinant monoclonal (MW7 epitope) for aggregate detection. Sequence-verified positive control. | View HTT Products |
| Validator | HTT siRNA Set (3 unique sequences). For allele-selective or total knockdown verification. | View HTT Products |
| Related Target: BDNF | Brain-Derived Neurotrophic Factor – neurotrophic support disrupted by mHTT. | View BDNF Products |
| Related Target: mTOR | Mechanistic Target of Rapamycin – autophagy-mediated clearance of mHTT aggregates. | View mTOR Products |
| Related Target: ATXN3 | Ataxin-3 – polyQ aggregation model, autophagy overlap. | View ATXN3 Products |
| Related Target: ATXN1 | Ataxin-1 – comparative polyQ disease target. | View ATXN1 Products |
| Related Target: TFEB | Transcription Factor EB – master regulator of autophagy-lysosome pathway. | View TFEB Products |
| Related Target: VCP | Valosin-containing protein – involved in protein clearance and aggresome formation. | View VCP Products |
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| Allele-selective mutant vs WT discrimination | Precise PolyQ length variants (Q23, Q73, Q103) with Mass Spec verified MW; WT (23Q) and Pathogenic (73Q/100Q) Exon 1 proteins with >95% purity. |
| Aggregate-prone protein handling & solubility | Endotoxin controlled (<1 EU/µg); low-endotoxin formulation prevents artifactual aggregation in neuronal assays. |
| Cross-species preclinical safety (Cyno/Mouse) | Human / Mouse / Cyno ortholog proteins available with sequence identity >95%. |
| Intracellular delivery validation | Ready-to-transduce Lentivirus particles for neuronal lineage stable expression. |
| Off-target specificity control | Validated siRNA included for HTT-specific vs non-specific toxicity differentiation. |
| Knockdown validation & controls | Validated siRNA included for HTT specificity checks and protein lowering validation. |
Live HTT R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
Global Clinical Landscape & Future Outlook
The therapeutic landscape for Huntington's disease (HD) is pivoting from non-selective gene silencing toward precision allele-selective strategies following the discontinuation of first-generation total HTT lowering therapies. Current R&D focuses on distinguishing mutant from wild-type huntingtin to preserve essential physiological functions while selectively clearing toxic polyQ-expanded proteins. Major players include Wave Life Sciences (allele-specific ASO WVE-003), uniQure (AAV-delivered microRNA AMT-130), and Novartis/PTC Therapeutics (small molecule splicing modulators). The next wave emphasizes mutant-specific protein clearance via PROTACs and autophagy enhancers targeting intracellular aggregate clearance.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| ASO / Gene Silencing | Roche/Ionis, Wave Life Sciences | Huntington's Disease | Allele-selective screening (Need Mutant vs WT proteins); knockdown validation (Need siRNA & lentivirus) |
| siRNA / RNAi | Alnylam, Arrowhead | Neurodegeneration | Intracellular uptake validation (Lentivirus stable lines) |
| Small Molecule (Splicing & Lowering) | Novartis (Branaplam), PTC Therapeutics | Huntington's Disease | Aggregation inhibition assays (High-purity mutant protein); splicing modulation assay |
| PROTAC / Targeted Degradation | Arvinas, Novartis | Huntington's Disease | Intracellular target engagement (Need stable cell line lentivirus) |
| Gene Therapy (AAV) | uniQure, Voyager Therapeutics | Huntington's Disease (CNS delivery) | Expression level standardization (Benchmark Abs); specificity validation (Need WT vs Mutant ORF) |
Key Mutations and Target Identity
The target requested (Huntingtin/HTT) resolves to the HTT gene (UniProt P42858). Key mutations reported include:
- D550 proteolytic cleavage inhibition: Mutation at residue D550 inhibits proteolytic cleavage and abolishes post-translational myristoylation (UniProt VAR_081737).
- LOMARS variant: dbSNP rs768047421, associated with a specific disease-related polymorphism.
- rs363075: A common SNP in the HTT gene (dbSNP rs363075).
These mutations underline the importance of allele-specific reagents and assays to discriminate between wild-type and mutant HTT.