In Vitro Efficacy Testing Services for Mitochondrial Disease
Drug R&D Solutions

In Vitro Efficacy Testing Services for Mitochondrial Disease

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We provide robust and sensitive in vitro screening and characterization platforms for accelerating the discovery and screening of potential therapies for Mitochondrial Disease. Our services offer targeted assays to evaluate compound efficacy, mechanism of action, and pathway modulation relevant to mitochondrial dysfunction. Key targets include enzymes and pathways involved in mitochondrial energy metabolism, oxidative stress, and electron transport chain integrity. We are equipped to assess associated pathological processes such as impaired ATP production, reactive oxygen species accumulation, and cellular bioenergetic deficits.

Our in vitro efficacy testing utilizes a range of biochemical and fluorescence-based assays to profile drug candidates targeting mitochondrial disease mechanisms. These methods enable precise quantification of enzyme activity, binding interactions, and pathway modulation. Collectively, our assays provide comprehensive data to support early-stage drug discovery and development.

3-hydroxyanthranilic acid as substrate: This method uses 3-hydroxyanthranilic acid to assess enzyme activity or metabolic pathway function, providing insights into mitochondrial biochemical processes.

Fluorescent assay: Fluorescent assays enable sensitive detection of enzymatic reactions or molecular interactions, allowing high-throughput screening of compound efficacy against mitochondrial targets.

Homogeneous Time Resolved Fluorescence (HTRF) assay: HTRF assays offer a robust, mix-and-measure approach for quantifying biomolecular interactions or protein levels, facilitating accurate assessment of target modulation in mitochondrial pathways.

We measure key pharmacological parameters to evaluate the potency and binding characteristics of drug candidates in our assays. These parameters provide critical information for ranking and optimizing compounds during lead identification and preclinical development. Accurate parameter measurement supports informed decision-making in the drug discovery pipeline.

IC-50: The concentration of a compound required to inhibit a specific biological function by 50%, serving as a standard indicator of drug potency.

Kb: The binding constant quantifying the affinity between a compound and its target, essential for understanding drug-target interactions and optimizing candidate selection.

Recommended In Vitro Efficacy Tests

5-Hydroxytryptamine Receptor 2B

The 5-Hydroxytryptamine Receptor 2B (5-HT2B) is implicated in mitochondrial disease pathogenesis and drug response. Testing 5-HT2B activity is crucial for identifying potential therapeutics and assessing off-target effects. Our service utilizes a sensitive Homogeneous Time Resolved Fluorescence (HTRF) assay to quantify receptor-ligand interactions. The primary parameter measured is the inhibition constant (Kb), enabling precise characterization of compound potency and selectivity in mitochondrial disease drug development.

Pharmacological Activity Material Method Parameter
Inositol phosphate turnover (serotonin-induced), inhibition CHO Chinese hamster ovary cells transfected with human 5-HT2B receptor Homogeneous Time Resolved Fluorescence (HTRF) assay Kb

Aminocarboxymuconate Semialdehyde Decarboxylase

Aminocarboxymuconate Semialdehyde Decarboxylase (ACMSD) regulates quinolinic acid levels, impacting mitochondrial health and neurodegeneration. ACMSD testing is vital for mitochondrial disease drug development, enabling evaluation of candidate inhibitors. Our assay uses 3-hydroxyanthranilic acid as substrate to measure enzyme activity. The primary parameter, IC50, quantifies inhibitor potency, supporting targeted therapeutic advancement for mitochondrial disorders.

Pharmacological Activity Material Method Parameter
2-amino-3-carboxymuconate-6-semialdehyde, inhibition Human enzyme 3-hydroxyanthranilic acid as substrate IC-50
2-amino-3-carboxymuconate-6-semialdehyde, inhibition Recombinant human enzyme 3-hydroxyanthranilic acid as substrate IC-50

Carnosine Dipeptidase 2

Carnosine Dipeptidase 2 (CNDP2) plays a critical role in mitochondrial function, and its dysregulation is linked to Mitochondrial Disease. CNDP2 testing is vital for drug development, enabling the identification of compounds that modulate its activity. Our service utilizes a sensitive fluorescent assay to measure enzyme activity, with IC-50 determination as the primary parameter, ensuring precise evaluation of inhibitor potency for therapeutic candidates.

Pharmacological Activity Material Method Parameter
Carnosine dipeptidase 2 (CNDP2), inhibition Purified enzyme Fluorescent assay IC-50
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