In Vitro Efficacy Testing Services for Fibrodysplasia Ossificans Progressiva

We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Fibrodysplasia Ossificans Progressiva (FOP). Our service offers comprehensive testing solutions to evaluate candidate compounds targeting key proteins such as ACVR1 (ALK2), BMP signaling components, and related pathways implicated in FOP. We specifically address the pathological processes of aberrant bone formation, inflammation, and dysregulated signal transduction. Our assays enable precise analysis of molecular and cellular mechanisms underlying ectopic ossification, supporting the development of targeted therapeutics.

Our in vitro efficacy testing encompasses a broad range of biochemical and cell-based assays, utilizing advanced detection technologies such as luminescence, fluorescence, and radioactivity. These methods are designed to assess compound activity, binding affinity, and pathway modulation relevant to FOP pathology. By employing diverse assay platforms, we provide comprehensive data on drug mechanism, potency, and selectivity.

ATP as substrate: Used in kinase assays to evaluate enzyme activity by measuring ATP consumption, which is critical for studying signaling pathways implicated in FOP.

ATP assay: Quantifies cellular ATP levels to assess cell viability, metabolic activity, or enzyme activity in response to test compounds.

Bioluminescence resonance energy transfer (BRET) assay: Detects real-time protein-protein interactions and pathway modulation in living cells, relevant for studying signaling networks in FOP.

Casein as substrate: Utilized in protease or kinase assays to monitor enzymatic modifications of casein, providing insights into enzyme specificity and activity.

Cell-based assay: Evaluates cellular responses, including proliferation, differentiation, and signaling changes, offering physiologically relevant data for FOP research.

Chemiluminescent assay: Measures biological activities or analyte concentrations with high sensitivity using chemiluminescent detection, suitable for low-abundance targets.

Competitive binding assay (qPCR): Assesses compound binding to target proteins using quantitative PCR readouts, useful for affinity and displacement studies.

Fluorescence resonance energy transfer (FRET) assay: Monitors molecular interactions or conformational changes by detecting energy transfer between fluorophores, aiding in pathway analysis.

Fluorescent assay: Employs fluorescent probes to quantify enzyme activity, cellular events, or biomarker levels with high sensitivity and specificity.

Gelatin as substrate: Applied in protease activity assays, especially for matrix remodeling enzymes, relevant to tissue and bone formation studies.

Homogeneous Time Resolved Fluorescence (HTRF) assay: A no-wash, high-throughput platform for quantifying biomolecular interactions and signaling events using energy transfer.

Luciferine/luciferase assay: Measures biological processes such as gene expression or cellular ATP by detecting light emission from luciferase-catalyzed reactions.

Radioactivity assay: Utilizes radiolabeled substrates to quantify enzyme activity or binding with high sensitivity and accuracy.

Retinoic acid assay: Evaluates the modulation of retinoic acid signaling, which may influence differentiation and ossification processes in FOP.

Transcription assay: Measures gene expression changes in response to therapeutic candidates, providing insights into pathway modulation and target engagement.

We measure key pharmacological parameters such as potency, affinity, and inhibitory capacity to characterize the efficacy of candidate compounds. These parameters inform on the strength and specificity of drug-target interactions and guide lead optimization. Accurate assessment of these variables is essential for advancing effective and selective FOP therapies.

EC-50: The concentration of a compound that produces 50% of its maximal effect, indicating potency and aiding in dose selection.

IC-50: The concentration of an inhibitor required to reduce a biological activity by 50%, used to assess compound efficacy against a target enzyme or pathway.

Kd: The dissociation constant representing the affinity between a ligand and its target, critical for evaluating binding strength and selectivity.

Ki: The inhibition constant that quantifies the binding affinity of an inhibitor for its target, important for comparing inhibitor effectiveness and mechanism.

Recommended In Vitro Efficacy Tests

Abl Proto-Oncogene 1, Non-Receptor Tyrosine Kinase

Abl Proto-Oncogene 1, Non-Receptor Tyrosine Kinase (ABL1) regulates signaling pathways implicated in aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). Testing ABL1 activity is crucial for identifying and optimizing drug candidates targeting pathological ossification. Key methods include kinase activity assays, phosphorylation analysis, and cell-based functional assays. Main parameters assessed are ABL1 phosphorylation status, kinase activity levels, and downstream signaling effects relevant to FOP pathology.

Activin A Receptor Like Type 1

Our Activin A Receptor Like Type 1 (ACVR1/ALK2) testing service supports Fibrodysplasia Ossificans Progressiva (FOP) drug development by targeting the mutated receptor driving abnormal bone formation. This testing enables the identification and characterization of novel inhibitors using advanced assays, including luciferase, FRET, BRET, qPCR, ATP, fluorescent, HTRF, and radioactivity-based methods. Key pharmacological parameters assessed include IC-50, Ki, and Kd, facilitating robust candidate evaluation and optimization.

Activin A Receptor Type 1

Activin A Receptor Type 1 (ACVR1) mutations drive aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). Precise ACVR1 testing is vital for FOP drug development to assess compound efficacy and binding. We offer assays including luciferase, qPCR-based competitive binding, ATP, FRET, fluorescence, cell-based, chemiluminescent, BRET, casein substrate, HTRF, and radioactivity. Key parameters—IC₅₀, Kᵢ, and Kd—enable robust evaluation of drug-target interactions.

Activin A Receptor Type 1B

Activin A Receptor Type 1B (ACVR1B) is implicated in aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). Testing ACVR1B activity is vital for developing targeted FOP therapies. Our service offers a comprehensive panel of assays—including luciferase, ATP, FRET, BRET, HTRF, fluorescent, cell-based, and radioactivity assays—to assess drug efficacy. Key parameters measured include IC₅₀ and Kd, providing critical data for inhibitor potency and binding affinity.

Alk Receptor Tyrosine Kinase

The Alk Receptor Tyrosine Kinase plays a pivotal role in aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). Testing for Alk activity is crucial for identifying therapeutic targets and assessing drug efficacy. Key methods include kinase activity assays and phospho-specific immunoassays. Main parameters measured are Alk phosphorylation levels, downstream signaling activation, and inhibition by candidate drugs. This service accelerates FOP drug development by providing precise, mechanism-based readouts.

Bone Morphogenetic Protein 6

Bone Morphogenetic Protein 6 (BMP6) plays a key role in aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). BMP6 testing is crucial for evaluating drug candidates targeting this pathway. Our service utilizes advanced methods including luciferin/luciferase, cell-based, chemiluminescent, HTRF, and transcription assays to assess BMP6 activity. IC50 values are determined to quantify drug efficacy, supporting the development of novel FOP therapeutics.

Bone Morphogenetic Protein Receptor Type 1A

Bone Morphogenetic Protein Receptor Type 1A (BMPR1A) plays a critical role in aberrant bone formation in Fibrodysplasia Ossificans Progressiva (FOP). Accurate BMPR1A testing is vital for FOP drug development, supporting inhibitor screening and efficacy assessment. We utilize robust methods including luciferase, ATP, FRET, fluorescence, chemiluminescence, cell-based, BRET, HTRF, radioactivity assays, and casein substrate analysis. Key parameter measured is IC-50, enabling precise evaluation of drug potency.

Bone Morphogenetic Protein Receptor Type 1B

Bone Morphogenetic Protein Receptor Type 1B (BMPR1B) is implicated in the pathological bone formation seen in Fibrodysplasia Ossificans Progressiva (FOP). Accurate BMPR1B testing is crucial for developing targeted FOP therapeutics. Our service utilizes advanced methods—including competitive binding (qPCR), ATP, FRET, fluorescent, BRET, HTRF, radioactivity assays, and casein substrate—to assess compound interactions. Key pharmacological parameters such as IC₅₀, Kᵢ, and K_d are determined to support drug discovery and optimization.

Cytochrome P450 Family 26 Subfamily B Member 1

Cytochrome P450 Family 26 Subfamily B Member 1 (CYP26B1) metabolizes retinoic acid, a key regulator implicated in Fibrodysplasia Ossificans Progressiva (FOP) pathogenesis. Testing CYP26B1 activity is vital for FOP drug development to assess compound efficacy and selectivity. Our service offers retinoic acid and radioactivity assays to measure CYP26B1 inhibition, providing IC-50 values as the main parameter for evaluating drug potency and optimizing candidate selection.

Fyn Proto-Oncogene, Src Family Tyrosine Kinase

The Fyn Proto-Oncogene, a Src Family Tyrosine Kinase, is implicated in aberrant signaling pathways driving heterotopic ossification in Fibrodysplasia Ossificans Progressiva (FOP). Testing Fyn activity is crucial for identifying effective FOP drug candidates. Our assay utilizes ATP as a substrate to evaluate compound inhibition, reporting key parameters including IC-50 and Ki values, thus supporting precise assessment of inhibitory potency and mechanism for FOP drug development.

Matrix Metallopeptidase 9

Matrix Metallopeptidase 9 (MMP-9) contributes to aberrant tissue remodeling in Fibrodysplasia Ossificans Progressiva (FOP). Testing MMP-9 activity is vital for evaluating candidate drugs targeting pathological ossification. Our assay uses gelatin as a substrate to measure MMP-9 enzymatic activity, providing IC-50 values to assess inhibitor potency. This enables precise screening of compounds for FOP drug development.

Receptor Interacting Serine/Threonine Kinase 2

Receptor Interacting Serine/Threonine Kinase 2 (RIPK2) influences inflammatory pathways implicated in Fibrodysplasia Ossificans Progressiva (FOP) progression. Testing RIPK2 activity is vital for evaluating therapeutic candidates targeting inflammation-driven heterotopic ossification. Key methods include kinase activity assays, Western blotting for phosphorylation status, and cell-based functional assays. Main parameters assessed are RIPK2 enzyme activity, phosphorylation levels, and downstream inflammatory signaling markers.

Ret Proto-Oncogene

Ret Proto-Oncogene testing assesses the involvement of RET signaling in Fibrodysplasia Ossificans Progressiva (FOP), a disorder marked by abnormal bone formation. This testing is crucial for identifying potential RET-targeted therapies. Using a Homogeneous Time Resolved Fluorescence (HTRF) assay, the service quantifies RET activity and determines drug potency by measuring IC-50 values, providing essential data for FOP drug development.

Retinoic Acid Receptor Gamma

Retinoic Acid Receptor Gamma (RARγ) modulates skeletal development and is a therapeutic target in Fibrodysplasia Ossificans Progressiva (FOP). RARγ testing is crucial for evaluating candidate drugs that may inhibit abnormal bone formation. Using luciferin/luciferase assays, this service quantifies RARγ activation by measuring luminescence. The main parameter assessed is EC-50, indicating compound potency and guiding drug optimization for FOP treatment.

Transforming Growth Factor Beta Receptor 1

Transforming Growth Factor Beta Receptor 1 (TGFβR1) plays a key role in the aberrant bone formation seen in Fibrodysplasia Ossificans Progressiva (FOP). Testing TGFβR1 activity is crucial for identifying and optimizing potential FOP therapeutics. Our service employs advanced methods—including luciferase, ATP, FRET, fluorescent, cell-based, BRET, casein-based, HTRF, and radioactivity assays—to measure main pharmacological parameters such as IC50 and Kd for effective drug development.