In Vitro Efficacy Testing Services

We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for iron overload diseases. Our service offers comprehensive in vitro efficacy testing specifically designed for drug candidates targeting key proteins and enzymes involved in iron metabolism and its associated pathological processes.

Our service employs a suite of advanced biochemical and cell-based assay methodologies tailored to the specific properties of each target. These methods include, but are not limited to:

• Enzyme-Linked Immunosorbent Assay (ELISA): For quantifying protein levels (e.g., target expression) or measuring specific protein-protein interactions.
• Luciferase Reporter Assays: To assess the impact of compounds on the transcriptional activity regulated by targets like HIF-2alpha or pathways influencing hepcidin expression.
• Fluorescence Resonance Energy Transfer (FRET) Assays: Ideal for studying protein-protein interactions, conformational changes, or enzyme activity in real-time.
• Chemiluminescence Analysis: A sensitive method applicable to various assay formats, including binding assays or reporter gene assays.
• Competitive Binding Assays: To determine the binding affinity of drug candidates to target receptors or transporters by competing with a known ligand.

Through these assays, we provide quantitative data on key pharmacological parameters essential for lead characterization and optimization, including:

• IC50 (Half Maximal Inhibitory Concentration): The concentration of a compound that inhibits a biological process by half.
• EC50 (Half Maximal Effective Concentration): The concentration of a compound that produces a half-maximal effect.
• Ki (Inhibition Constant): A measure of the affinity of an inhibitor for an enzyme.
• Kd (Dissociation Constant): A measure of the binding affinity between a molecule and its target.
• MEC (Minimum Effective Concentration): The lowest concentration of a drug that produces a desired effect.
• MIC (Minimum Inhibitory Concentration): (Typically used for antimicrobial agents, but can be adapted for cell viability assays if iron toxicity or ferroptosis is being inhibited).

Our experienced team specializes in developing robust, reproducible, and high-throughput compatible assays to support your drug discovery pipeline from initial screening to lead optimization. We are committed to providing high-quality data to help you identify promising drug candidates and understand their mechanism of action against targets relevant to iron overload.

Partner with us to accelerate your preclinical assessment of novel iron overload therapeutics.

Recommended In Vitro Efficacy Tests

Dihydroorotate Dehydrogenase (DHODH)

Investigating potential links between pyrimidine synthesis, mitochondrial function, and iron-mediated cell death pathways like ferroptosis.

Endothelial PAS Domain Protein 1 (EPAS1 / HIF-2alpha)

Evaluating drug effects on a key transcription factor regulating the expression of iron transporters such as DMT1 and Ferroportin, particularly relevant for intestinal iron absorption.

Glutathione Peroxidase 4 (GPX4)

Assessing the protective effects of drug candidates against ferroptosis by modulating the activity or expression of this essential anti-ferroptotic enzyme.

Solute Carrier Family 11 Member 2 (SLC11A2 / DMT1)

Characterizing the impact of compounds on the function or expression of this crucial transporter responsible for cellular iron uptake.

Solute carrier Family 40 Member 1 (SLC40A1 / Ferroportin)

Evaluating the modulatory effects of drug candidates on the activity or regulation of the primary cellular iron exporter, a key target for controlling systemic iron levels.

ST14 Transmembrane Serine Protease Matriptase (ST14)

While TMPRSS6 (Matriptase-2) has a more established role in systemic iron regulation via hepcidin, we can explore potential roles or off-target effects related to other matriptases like ST14.

Synuclein Alpha (SNCA)

Investigating the interaction between drug candidates and alpha-synuclein, particularly relevant for understanding and mitigating iron-related neurotoxicity and protein aggregation observed in certain conditions.

Transferrin Receptor (TFRC)

Analyzing the influence of compounds on the binding of transferrin to its receptor or the overall process of receptor-mediated iron uptake into cells.

Transmembrane Serine Protease 6 (TMPRSS6)

Assessing the impact of drug candidates on this key negative regulator of hepcidin expression, crucial for modulating systemic iron levels.