In Vitro Efficacy Testing Services for Primary Sclerosing Cholangitis

We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Primary Sclerosing Cholangitis (PSC). Our services are tailored to assess compound efficacy and mechanism of action in cellular and molecular systems relevant to PSC, enabling high-confidence decision-making in early drug development. Key targets and pathways include bile acid transporters, nuclear receptors, and inflammatory mediators implicated in cholestasis and fibrotic progression. Our assays are designed to evaluate compound impact on bile acid metabolism, hepatocellular function, and inflammatory processes central to PSC pathology.

We offer a comprehensive suite of biochemical and cell-based assays to evaluate the pharmacological activity of candidate compounds in PSC models. These methods enable quantification of pathway modulation, transporter activity, gene expression, and functional cellular responses relevant to disease mechanisms. Collectively, our approaches support robust efficacy profiling and mechanistic insights.

Chemiluminescent assay: Utilizes enzyme-catalyzed light emission to detect and quantify target molecules or pathway activity with high sensitivity, supporting rapid screening of compound effects.

Gene reporter assay: Employs genetically engineered cells expressing luminescent or fluorescent reporters under control of disease-relevant promoters to monitor transcriptional regulation in response to test agents.

Homogeneous Time Resolved Fluorescence (HTRF) assay: Measures molecular interactions or signaling pathway activation using fluorescence resonance energy transfer, enabling high-throughput quantification of biomolecular events.

Luciferine/luciferase assay: Monitors cellular events such as gene expression or viability through bioluminescent signals, providing sensitive readouts for pathway engagement.

RNA assay: Quantifies changes in gene expression (e.g., upregulation or suppression of target transcripts) following compound treatment to elucidate molecular mechanisms.

SRC-2-2 peptide recruitment assay: Evaluates nuclear receptor modulation by measuring recruitment of co-regulator peptides, informing on the functional impact of compounds on transcriptional machinery.

Taurocholate uptake assay: Assesses the activity of bile acid transporters by measuring cellular uptake of labeled taurocholate, directly relevant to PSC-associated cholestasis.

We measure key pharmacological parameters such as potency, efficacy, and inhibitory concentrations to comprehensively characterize compound activity. These metrics are essential for comparing candidate molecules and informing dose selection in subsequent studies. By quantifying parameters like EC-50, IC-50, MEC, and MIC, we provide actionable data for advancing promising PSC therapeutics.

EC-50 (Half maximal effective concentration): Indicates the concentration of a compound required to achieve 50% of its maximal effect, reflecting compound potency.

IC-50 (Half maximal inhibitory concentration): Represents the concentration needed to inhibit a specific biological activity by 50%, critical for assessing antagonist or inhibitor efficacy.

MEC (Minimum effective concentration): The lowest concentration at which a compound produces a measurable desired effect, important for identifying effective dose ranges.

MIC (Minimum inhibitory concentration): The lowest concentration required to inhibit a specific biological or cellular process, often used to gauge compound selectivity and safety margins.

Recommended In Vitro Efficacy Tests

Atp Binding Cassette Subfamily B Member 11

The Atp Binding Cassette Subfamily B Member 11 (ABCB11) is crucial for bile acid transport, and its dysfunction contributes to bile duct injury in Primary Sclerosing Cholangitis (PSC). Testing ABCB11 activity, particularly using chemiluminescent assays, is vital for evaluating drug effects in PSC drug development. The assay quantifies transport activity, with MEC (Minimum Effective Concentration) as a key parameter, enabling precise assessment of candidate drug efficacy.

Atp Binding Cassette Subfamily B Member 4

The Atp Binding Cassette Subfamily B Member 4 (ABCB4) plays a vital role in bile phospholipid transport, and its dysfunction is implicated in Primary Sclerosing Cholangitis (PSC). Testing ABCB4 activity is crucial for evaluating drug candidates targeting PSC. Our service utilizes a chemiluminescent assay to measure ABCB4-mediated substrate transport, with MEC (Minimum Effective Concentration) as the primary parameter, ensuring precise assessment of drug efficacy in modulating ABCB4 function.

C-C Motif Chemokine Receptor 2

The C-C Motif Chemokine Receptor 2 (CCR2) is implicated in the inflammatory pathogenesis of Primary Sclerosing Cholangitis (PSC). CCR2 testing is crucial for evaluating therapeutic targets and drug efficacy in PSC drug development. Our service utilizes sensitive RNA assays to quantify CCR2 expression, with Minimum Inhibitory Concentration (MIC) as a main parameter to assess compound potency, enabling informed decision-making in preclinical research.

C-C Motif Chemokine Receptor 5

C-C Motif Chemokine Receptor 5 (CCR5) plays a key role in the inflammatory processes of Primary Sclerosing Cholangitis (PSC). CCR5 testing is crucial for evaluating therapeutic targets and drug efficacy in PSC drug development. Our service uses sensitive RNA assays to quantify CCR5 expression, with Minimum Inhibitory Concentration (MIC) as a main parameter to assess compound potency, supporting informed decision-making in early-stage PSC research.

Nuclear Receptor Subfamily 1 Group H Member 4

Our Nuclear Receptor Subfamily 1 Group H Member 4 (NR1H4/FXR) testing service supports Primary Sclerosing Cholangitis drug development by evaluating compounds targeting FXR, a key regulator of bile acid homeostasis implicated in disease progression. Using SRC-2-2 peptide recruitment, HTRF, and luciferin/luciferase assays, we assess compound efficacy and potency. Main parameters measured include EC50, IC50, and MEC, enabling precise optimization of candidate therapeutics.

Peroxisome Proliferator Activated Receptor Alpha

Peroxisome Proliferator Activated Receptor Alpha (PPARα) modulates bile acid metabolism and inflammation in Primary Sclerosing Cholangitis (PSC). Testing PPARα activation is crucial for PSC drug development to assess therapeutic efficacy and safety. Our service utilizes gene reporter and luciferin/luciferase assays to quantitatively measure PPARα activation, providing precise EC-50 values for candidate compounds, enabling efficient compound screening and optimization in PSC research.

Peroxisome Proliferator Activated Receptor Delta

Peroxisome Proliferator Activated Receptor Delta (PPARδ) is implicated in modulating inflammation and fibrosis in Primary Sclerosing Cholangitis (PSC). Testing PPARδ activity is crucial for evaluating potential PSC therapeutics. Our service employs gene reporter and luciferin/luciferase assays to quantitatively assess PPARδ activation. The main parameter measured is EC-50, enabling precise determination of compound potency and efficacy in activating PPARδ for PSC drug development.

Platelet Derived Growth Factor Receptor Beta

Platelet Derived Growth Factor Receptor Beta (PDGFR-β) is implicated in hepatic fibrogenesis in Primary Sclerosing Cholangitis (PSC). Testing PDGFR-β expression and activity is crucial for assessing anti-fibrotic drug efficacy. Key methods include immunohistochemistry, Western blotting, and qPCR. Main parameters analyzed are PDGFR-β protein/mRNA levels, phosphorylation status, and downstream signaling activity, providing essential data for PSC drug development targeting fibrotic pathways.

Solute Carrier Family 10 Member 2

Solute Carrier Family 10 Member 2 (SLC10A2) regulates bile acid transport, playing a crucial role in Primary Sclerosing Cholangitis (PSC) pathophysiology. Testing SLC10A2 function is vital for PSC drug development to assess compound efficacy and safety. Our service utilizes the taurocholate uptake assay to measure SLC10A2 activity, determining the IC-50 values of candidate drugs, thereby enabling precise evaluation of their inhibitory potential on bile acid transport.