Target Validation
BOC Sciences' target verification platform can verify a large number of potential drug targets for customers, and provide R&D technical support in the fields of tumors, metabolic diseases, and pharmacogenomics.
Why is target validation needed?
The process of new drug development is complex and lengthy. The discovery and validation of drug targets is the key cornerstone of the successful development of new drugs. Target validation can be chemical or genetic. The former means that the therapeutic effect can be achieved by regulating the target protein with chemical substances. The latter means that the therapeutic effect is achieved through genetic technology. Target confirmation is also divided into clinical and preclinical. If the compound that regulates a target shows efficacy in clinical trials, the researchers will start the project development. This strategy no longer makes commercial sense. Therefore, the current target validation is mainly preclinical validation.
Target validation at an early stage increases the understanding between target manipulation and disease treatment effects, thereby increasing the success rate of clinical trials. Once a candidate target passes validation experiments, the project enters the hit identification phase.
Steps in Target Validation
The drug targets obtained by multiple target identification methods can only be used as a method for preliminary screening of targets, and then the candidate targets need to be verified.
- Step 1: Confirm whether the candidate target modulates the biological activity of the compound in cell experiments. This process can be accomplished by techniques such as target gene overexpression, target gene silencing, and target gene knockout.
- Step 2: Quantify the affinity of small molecules and targets. This process can be accomplished by surface plasmon resonance technology, isothermal calorimetry and other means.
- Step 3: Use NMR or co-crystallization experiments for more rigorous verification, and then obtain the 3D structure of the drug-target complex.
Why choose us?
Target verification is the first and crucial step in new drug discovery. BOC Sciences' target validation service aims to provide customers with strong R&D support in identifying and validating the relevance of known or new targets to specific diseases, helping customers make decisions on whether to advance the project, thereby effectively reducing the risk of drug research and development projects.
Our services
- Bioinformatics Support
There is not a simple one-to-one correspondence between diseases and targets, but an intricate network of associations is formed between them. Through systematic bioinformatics analysis, we screen out the targets related to the selected diseases in the complex disease network and verify them experimentally.
- In Vitro Disease Model
To investigate disease mechanisms, target validation studies are performed using various genetic or genomic approaches in in vitro cell lines, animal disease models, and human disease models.
- Suitable cell lines
- 3D culture and co-culture models
- Human Stem Cells (iPSCs)
- Functional/pharmacogenomics
We apply techniques such as genomics, gene silencing strategies, gene knockout strategies, standard genetics, and chemical genetics to perturb relevant in vitro systems to demonstrate target efficacy.
- Target gene silencing based on siRNA technology
- Target gene silencing based on shRNA technology
- target gene overexpression
- Target gene knock-in/knock-out
- Gene editing
- Functional verification of target genes in vitro and in vivo
- Biomarkers
Through the analysis, identification and verification of biomarkers through transcriptomics, proteomics, flow cytometry and other technologies, we can accurately find the cause of the disease and the target of treatment.