Market Intelligence, Clinical Progress, and High-Purity Reagents for Neurodegenerative, Autoimmune, and Antiviral Development.
TarMart Solution Ecosystem & Related Targets
Comprehensive reagent toolkit for PIP5K3/PIKFYVE drug discovery. Select your modality below:
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen | PIKFYVE Recombinant Protein (Kinase Domain aa 1358-2068) / Full Length Protein. High purity (>95%), Endotoxin <1EU/ug. Sequence Verified. Theoretical MW confirmed by MS. | View PIKFYVE Products |
| Gene Delivery | PIKFYVE Promise-ORF / Lentivirus Premade Particles. Full-length ORF (NM_001018112) for stable cell line construction in neuronal or HEK293 systems. Endotoxin controlled. | View PIKFYVE Products |
| Benchmark Ab | Anti-PIKFYVE Antibody. Recombinant positive control for western blot / IHC. | View PIKFYVE Products |
| Benchmark Control | Catalytic Dead (D1702A) Mutant Protein. Kinase-dead negative control for specificity assays. Sequence verified. | View PIKFYVE Products |
| Validator | PIKFYVE siRNA Set (3 unique sequences). For knockdown verification in autophagy flux, endosomal trafficking, and functional assays. | View PIKFYVE Products |
| Related Target A | MTOR. Autophagy and lysosomal regulation pathway partner. | View MTOR Products |
| Related Target B | BECN1. Crucial for autophagosome formation and PI3K complex regulation. | View BECN1 Products |
| Related Target C | FIG4 Phosphatase Protein. Functional antagonist regulating PI(3,5)P2 lipid levels; synthetic lethal screening partner. | View FIG4 Products |
| Related Target D | VAC14 Scaffold Protein. Essential complex component for PIP5K3 activation; ALS/FTD genetic link. | View VAC14 Products |
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| Kinase Selectivity (vs PIK3 family, ATP-competitive vs allosteric) | Purified active kinase domain (>95%) with native folding; mass spec-verified for binding mode validation. |
| Lipid Substrate (PI(3)P) Interference | Full-length & FYVE-domain constructs for substrate competition assays. |
| Neuronal Cell Model Construction | Lentivirus with CMV/EF1a promoters for SH-SY5Y/iPSC-derived neuron transduction. |
| Lack of Proper Controls | Sequence-verified reagents, catalytic dead mutant (D1702A), and validated siRNA + ORF rescue package. |
| Cellular Phenotype Reproducibility | Lentivirus-driven stable overexpression with native processing. |
Live PIKFYVE R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
- ➤ View Active Clinical Trials (general)
- ➤ View Active ALS Clinical Trials (PIKFYVE-related)
- ➤ Latest Autophagy Modulation Research
- ➤ Latest Resistance Research
- ➤ Recent Patent Filings
Global Clinical Landscape & Future Outlook
The race for PIP5K3/PIKFYVE-targeted therapeutics is intensifying, with major players shifting focus from early oncology, broad immunosuppression, and antiviral screening to neurodegenerative diseases, particularly ALS and FTD. First-generation small molecule inhibitors like Apilimod (ATP-competitive) established the pharmacological foundation but faced lysosomal toxicity limitations (lamellar body accumulation). The next wave of R&D targets highly selective CNS-penetrant inhibitors, allosteric modulators, and PROTACs that can modulate endolysosomal trafficking without inducing severe on-target toxicity. Genetic validation from C9orf72, GRN, and SPG11 carriers underscores a therapeutic window for partial inhibition.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| Small Molecule Inhibitor (ATP-competitive) | AI Therapeutics, legacy pharma | ALS, Autoimmune, Oncology | Enzymatic Assay (Need high-purity Kinase Domain) |
| Small Molecule (Allosteric) | Maze Therapeutics, Academic Consortia | ALS, FTD (C9orf72, GRN carriers) | Conformation-specific binding assay (Need mutant vs WT full-length protein) |
| Covalent Inhibitor | Stanford University (published scaffold) | Viral entry (Ebola, SARS-CoV-2) | Cysteine accessibility assay (Need pure kinase domain with intact Cys residues) |
| PROTAC / Degrader | Emerging Biotechs | Neurodegeneration, Oncology (autophagy addiction) | Degradation tracking (Need Benchmark Abs & Lentivirus models); Binary/ternary complex formation (Need high-quality protein for SPR) |
| siRNA / Antisense / Genetic Modulation | Academic/Translational, ASO developers | Viral infection entry, SPG11-related HSP | Knockdown validation (Need accurate siRNA sets); Potency comparison (Need validated siRNA controls) |