Target validation is a process to accumulate the evidence for and against a target, and drug developers typically spend several months conducting new research or replicating literature studies to build sufficient evidence. As an industry-leading biopharmaceutical company, Creative Biolabs provides comprehensive target validation and qualification services for psychiatric disorders to global customers.
Importance of Target Validation and Qualification
The need for novel therapies for central nervous system (CNS) disorders with improved efficacy, safety, and tolerability has never been in question, as it is widely recognized that, even with currently utilized treatments, CNS disorders are major contributors to the global burden of illness and incur high economic costs. However, during the development of drugs for CNS disorders, the core of the problem lies in the identification and validation of novel drug targets for these disorders.
Target Validation and Qualification
To validate the target in pharmacology, first, whether the target protein is expressed and active or not in the desired organ/subregion/cell types will be validated. In the next stage, target RNA and protein expression altered by the disease will be detected. There are three metrics in the target qualification: 1) A pharmacological tool (e.g., a small molecule, antibody, or peptide) modulates disease associated with a pathway in vitro or in heterologous cell lines at appropriate concentrations. 2) Ligands with the intended mode of action modulate disease-associated pathways ex vivo or in native tissue, or in the best case. 3) Ligands with the intended mode of action modulate disease-associated pathways in vivo and target engagement–activity relationships are established.
There are several stages to validate the target in genetically engineered models. First, genetic association with disease occurs in small, underpowered, or non-replicated studies. Second, polygenic association with modest effect size and known function of the variant, or association with a common, low-risk variant in a gene that also has rare variants associated with large effect size, or in the best case. Third, monogenic association with large effect size and known function of the gene variant. To qualify the target in genetically engineered models, usually genetic modulation first in a non-mammalian model organism, and then rodent/nonhuman primate produces a disease- or treatment-relevant phenotype.
In this validation point, clinically relevant efficacy is first observed in a small trial and then observed with at least one ligand with a different mode of target modulation or with two ligands on biomarkers previously shown to predict efficacy. Finally, the validation needs at least one ligand with an analogous mode of action on the target/target pathway that has “approvable” efficacy in the indication of interest and robust evidence of target engagement.
Services at Creative Biolabs
Committed to psychotropic drug development, Creative Biolabs has extensive experience accumulated from hundreds of successful projects in target validation and qualification services. We have established an excellent platform with comprehensive technologies and professional specialists to provide reliable target validation and qualification services.
As previously mentioned, target validation and qualification play a crucial role in psychotropic drug development. Within this context, Creative Biolabs, an industry-leading biopharmaceutical company focused on psychiatric disorders has certainly accumulated extensive experience in relative services. If you have any requirements or questions in target validation and qualification, please feel free to contact us for more information.
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