In Vitro Efficacy Testing Services for Cystic Fibrosis

We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Cystic Fibrosis. Our services enable detailed evaluation of compound efficacy, mechanism of action, and potency in models relevant to Cystic Fibrosis research. Key molecular targets include the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein and associated ion channels and signaling pathways involved in epithelial ion transport. We assess pathological processes such as defective chloride ion transport, dysregulated airway surface liquid, and impaired mucociliary clearance.

Our comprehensive suite of in vitro assays encompasses biochemical, cellular, and electrophysiological techniques to evaluate the pharmacological activity of candidate compounds. These methods are designed to quantify binding, signaling, and functional effects, supporting both early screening and detailed mechanistic studies.

Chemiluminescent assay: Utilized to measure enzyme activity or cellular responses by detecting light emission, enabling sensitive quantification of biological processes relevant to CFTR function.

Displacement of [3H]-DPCPX: A radioligand binding assay used to assess compound binding affinity to specific receptors or proteins, supporting target validation and competitive binding studies.

ELISA assay: Enzyme-linked immunosorbent assay applied to detect and quantify proteins, cytokines, or biomarkers associated with Cystic Fibrosis, facilitating assessment of therapeutic impact.

Fluorescent assay: Employs fluorescence-based detection for real-time analysis of ion transport, protein interactions, or signaling pathways, providing high sensitivity in functional studies.

Patch-clamp assay: An electrophysiological technique to directly measure CFTR channel activity and ion currents in cells, offering precise functional characterization of compounds.

Short-circuit current assay: Used to quantify transepithelial ion transport in epithelial cell monolayers, directly reflecting CFTR-mediated chloride secretion and compound efficacy.

We measure key pharmacological parameters such as EC-50 and Ki to quantitatively evaluate compound potency and binding affinity. These metrics are essential for comparing candidate molecules and guiding lead optimization in drug development for Cystic Fibrosis.

EC-50: The concentration of a compound required to achieve half-maximal effect, serving as a standard indicator of compound potency in functional assays.

Ki: The equilibrium dissociation constant for inhibitor binding, reflecting the affinity of a compound for its target and supporting structure-activity relationship studies.

Recommended In Vitro Efficacy Tests

Adenosine A1 Receptor

The Adenosine A1 Receptor is implicated in airway inflammation and mucus secretion in Cystic Fibrosis. Testing its activity is crucial for evaluating potential therapeutics. Our service measures ligand binding affinity via displacement of [3H]-DPCPX, a selective A1 antagonist, allowing precise determination of Ki values. This provides essential data for drug candidate screening and optimization in Cystic Fibrosis drug development.

Cf Transmembrane Conductance Regulator

The CF Transmembrane Conductance Regulator (CFTR) is a chloride channel whose dysfunction causes Cystic Fibrosis. CFTR testing is vital in drug development to assess compound efficacy in restoring channel function. Key methods include short-circuit current, chemiluminescent, fluorescent, ELISA, and patch-clamp assays. These techniques measure parameters such as EC-50, indicating compound potency, to guide therapeutic optimization and patient-specific treatment strategies.