Market Intelligence, Clinical Progress, and High-Purity Reagents for Primary Hyperoxaluria Therapeutics Development.
HAO1 (glycolate oxidase) is an FMN-dependent hydroxy acid dehydrogenase expressed primarily in the liver, making it a key therapeutic target for Primary Hyperoxaluria (PH).
TarMart Solution Ecosystem & Related Targets
Comprehensive reagent toolkit for HAO1 drug discovery. Select your modality below:
| Component / Network | Product Description | Product Link |
|---|---|---|
| Antigen | HAO1 Recombinant Protein – Full-length soluble oxidoreductase, FMN cofactor compatible, >95% purity, Endotoxin <1EU/μg. Sequence Verified, theoretical MW confirmed. | View HAO1 Products |
| Gene Delivery | HAO1 Lentivirus Premade Particles – ORF-based overexpression for HepG2/stable liver cell lines. High titer (>10^8 TU/mL), Endotoxin controlled (<1EU/μg). | View HAO1 Products |
| Validator (siRNA) | HAO1 siRNA Set (3 target-specific + 1 control) – For knockdown verification and assay specificity controls. Chemically synthesized, HPLC purified. | View HAO1 Products |
| Ortholog Panel | HAO1 Cynomolgus/Mouse/Rat Ortholog Proteins – Cross-species sequence verified for toxicology and PK/PD bridging studies. | View HAO1 Products |
| Benchmark Antibody | Anti-HAO1 Antibody – Recombinant positive control for western blot and ELISA, specific for HAO1 protein quantification. | View HAO1 Products |
| Related Target: LDHA | LDHA – Synergistic pathway target for Primary Hyperoxaluria therapies. | View LDHA Products |
| Related Target: AGXT | AGXT (Alanine-Glyoxylate Aminotransferase) – Defective in PH1; upstream metabolic node for combination strategy. | View AGXT Products |
| Related Target: GRHPR | GRHPR (Glyoxylate Reductase/Hydroxypyruvate Reductase) – Defective in PH2; complementary pathway analysis. | View GRHPR Products |
| Related Target: HOGA1 | HOGA1 (4-Hydroxy-2-Oxoglutarate Aldolase) – Defective in PH3; pan-PH therapeutic strategy relevance. | View HOGA1 Products |
Critical Assay Challenges and TarMart Advantage
| Critical Assay Challenge | The TarMart Advantage (Technical Spec) |
|---|---|
| Enzymatic Assay Standardization & Activity Preservation | High purity (>95%) recombinant HAO1 expressed in HEK293 with intact FMN-binding domain; confirmed by spectrophotometric activity (glycolate to glyoxylate conversion). |
| RNAi Knockdown Validation | Highly specific Anti-HAO1 antibodies for precise protein-level quantification; combined with validated siRNA set for target specificity. |
| Lack of Controls | Benchmark antibodies included for assay standardization. |
| Liver-Specific Delivery Assays | Ready-to-use Lentivirus for stable HepG2/PHH cell line construction; Endotoxin controlled (<1EU/μg) for sensitive hepatocyte cultures. |
| Pan-PH Strategy Validation (Types 1/2/3) | Full metabolic pathway panel: HAO1 + AGXT + GRHPR + HOGA1 recombinant proteins available for cross-reactivity and combination screening. |
| False Positives in HTS | siRNA validation set included for target-specificity confirmation; Sequence-verified shRNA alternative available. |
Live HAO1 R&D Tracker
Market data changes daily. Access the latest global pipeline status directly:
Global Clinical Landscape & Future Outlook
The race for HAO1-targeted therapeutics has shifted from rare disease validation to modality expansion. Following the approval of Lumasiran (siRNA, Alnylam) and Nedosiran (siRNA, Dicerna/Ionis) for Primary Hyperoxaluria, the next wave of R&D focuses on oral small molecule inhibitors (Vertex, Allena) and permanent genetic correction via base editing (Beam Therapeutics). As first-generation RNAi therapies demonstrate liver-specific HAO1 knockdown efficacy, differentiation now centers on pan-PH coverage (Types 1, 2, and 3), patient compliance (subcutaneous vs. oral), and addressing non-responder populations through metabolic pathway combination approaches.
Competitive Modality & Indication Snapshot
| Modality | Representative Players | Key Indications | Critical Assay Need (Why TarMart?) |
|---|---|---|---|
| RNAi (siRNA) | Alnylam (Lumasiran/Oxlumo), Dicerna/Ionis (Nedosiran/Rivfloza) | PH Type 1, 2, 3 | Liver Cell Uptake Assays (Need Lentivirus-stable HepG2 lines for uptake validation) |
| Small Molecule Inhibitors | Vertex Pharmaceuticals, Allena Biomedical | PH Type 1 (Oral alternative) | Enzymatic Activity Assay (Need active recombinant HAO1 with cofactor binding site integrity) |
| Gene Therapy (Adeno-associated virus) | Academic Spin-offs | Genetic Liver Diseases | Expression Validation (Need specific antibodies) |
| Gene Editing (Base Editing) | Beam Therapeutics | Severe PH1 | Knockdown Validation (Need siRNA controls and stable overexpression lines for editing efficiency quantification) |
| mRNA Therapy | Moderna (preclinical), Beam | AGXT-deficient PH1 | Expression Kinetics (Need high-purity antigen standards for ELISA quantification) |