Target Identification and Validation in Drug Discovery

Application Background

One of the most important steps in developing a new drug is target identification and validation.  Target-based drug discovery has become the prevailing paradigm used by pharmaceutical and biotechnology companies. This approach is appealing because it holds the promise of identifying more efficacious compounds with fewer undesirable side effects. Good target identification and validation enables increased confidence in the relationship between target and disease and allows the exploration of whether target modulation will lead to mechanism-based side effects.

Drug Target Identification

Drug Target Identification is a first step for improving efficacy, tracing and avoiding side-effects as well as understanding the mode-of-actions of drug candidates.

Drug Target Validation

Target validation refers to the accumulation of scientific evidence that a target (usually a protein) is regulated (usually inhibited) and has therapeutic uses. There are several implications here. Target validation can be chemical or genetic validation. The former means that the therapeutic effect can be achieved through the regulation of the target protein by chemical substances, and the latter means that the therapeutic effect can be produced by genetic technology such as animal gene knockout or human gene mutation. Target validation is also divided into clinical and pre-clinical. If a compound that modulates a target shows efficacy in clinical trials, and you start the project, it is the so-called me-too research and development, which is no longer commercially meaningful. However, people with gene activation and inactivation mutations like PCSK9 exist, and the signs are opposite (a high blood lipid and a low blood lipid, the cardiovascular risk is the opposite), and no other abnormal targets are rare. Therefore, the current target validation is mainly pre-clinical validation, that is, animal experiment verification.

The different between Target Validation:

Target Validation: The process of demonstrating in a clinical trial that engaging the target provides statistically meaningful therapeutic benefit with acceptable safety for a given indication.

Target Qualification: Preclinical or limited clinical studies prior to well powered clinical trials, that establish the scientific validity and safety of a drug target; it is part of the continuum of target validation.

Target Identification: The generation of scientific evidence that a manipulatable target is involved in some significant way in a disease process.

New target validation is the basis of completely new drug exploration and the initial step of drug discovery.  New drug target validation might be of great help not only to new drug research and development but also provide more insight into the pathogenesis of target related diseases. Basically, the target validation process might include six steps:
1. Discovering a biomolecule of interest.
2. Evaluating its potential as a target.
3. Designing a bioassay to measure biological activity.
4. Constructing a high-throughput screen.
5. Performing screening to find hits.
6. Evaluating the hits.

This service was designed to meet your specific needs fast at extremely competitive prices. We can also provide screening services on specified signaling pathways areas in accordance to your needs,by screening only a subset of the library you can use this service at a reduced price.
With several years of preclinical research expertise and experience, Medicilon’s Target Identification and Validation Services provide a wide range of RNAi research services to companies and research institutions worldwide. Our research and technical support teams ensure the top-quality products and services to meet the unique needs of clients. Our world-class teams have extensive nucleic acid therapeutic manufacturing and development expertise.

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