Target Identification and Validation Service

Target identification and validation are critical steps in drug discovery, focusing on identifying molecular targets associated with disease processes and confirming their biological and therapeutic relevance. This process ensures that selected targets play a pivotal role in disease mechanisms, enabling the development of precise and effective therapies. By combining advanced techniques such as genomic and proteomic profiling, functional assays, CRISPR/Cas9 gene editing, and in vivo validation models, target identification and validation provide a comprehensive understanding of target functionality, mechanism of action, and potential for therapeutic intervention. These steps address key challenges in drug development, including poor efficacy, off-target effects, and limited insight into complex disease pathways. 

 

At MtoZ Biolabs, we specialize in providing comprehensive and customizable target identification and validation service to accelerate drug discovery and development. With a multidisciplinary team and advanced technological platforms, we aim to streamline the discovery of therapeutic targets and ensure their biological relevance for further research and therapeutic applications.

 

1. Target Identification

Our target identification and validation service integrates advanced methodologies to uncover disease-associated molecules effectively. We leverage cutting-edge genomic and transcriptomic analysis, including next-generation sequencing and expression profiling, to identify genetic variations and molecular pathways involved in disease mechanisms. Additionally, we employ phenotypic screening with high-content imaging and advanced cellular assays to reveal critical targets based on functional changes in disease models. Our bioinformatics capabilities further support a comprehensive understanding of molecular interactions and regulatory networks.

 



Liu, M. et al. Sci Rep. 2021.

Figure 1. Basic Workflow of Target Identification

 

2. Target Validation  

Once potential targets are identified, we rigorously validate their biological and therapeutic relevance. Using technologies like CRISPR/Cas9 and RNA interference, we precisely manipulate target expression to assess their roles in disease progression. Proteomic analyses and functional assays further confirm the involvement of target molecules in key cellular processes. For more complex evaluations, we utilize animal models to study the in vivo effects of target modulation, ensuring robust validation for therapeutic applications.

 

Service Advantages 

1. Tailored Solutions: Our target identification and validation service is customized to fit the specific needs of your research, ensuring effective and focused outcomes.  

2. Advanced Technology: We employ state-of-the-art platforms for accurate target identification and validation.  

3. Experienced Team: Our scientists have extensive expertise in drug discovery, ensuring reliable and actionable results.  

 

Case Study

Case 1. This study identified the phytochemical hyperforin (HPF) as an anti-obesity agent using the Connectivity Map (CMap) approach. HPF promotes thermogenesis in adipose tissue by activating AMPK and PGC-1α through a Ucp1-dependent pathway. Using LiP-SMap, microscale thermophoresis assays, and molecular docking analysis, dihydrolipoamide S-acetyltransferase (Dlat) was confirmed as a direct molecular target of HPF. Ablation of Dlat significantly reduced HPF-induced adipose tissue browning both in vitro and in vivo. Furthermore, genome-wide association studies revealed a significant correlation between DLAT variation and human obesity, highlighting its potential as a therapeutic target. Our target identification and validation service applies advanced techniques, such as proteomics-based profiling and functional assays, to uncover and validate molecular targets, expediting the development of therapies for metabolic diseases like obesity.

 



Chen, S. et al. Cell Metab. 2021.

Figure 2. Identification of Potential HPF Targets Using LiP-SMap

 

Case 2. This study identified a promising natural small molecule, daphnepedunin A (DA), through a bioassay-guided chemical investigation of the medicinal plant Wikstroemia chamaedaphne. DA demonstrated significant anti-fibrotic effects both in cultured renal fibroblasts and in a unilateral ureteral obstruction (UUO) mouse model, outperforming the clinical trial drug pirfenidone. Using thermal proteome profiling (TPP), cell division cycle 42 (Cdc42) was identified as the direct target of DA. Mechanistically, DA inhibits Cdc42 activity, downregulates its downstream signaling molecules p-PKCζ and p-GSK-3β, and promotes the phosphorylation and ubiquitin-dependent degradation of β-catenin at Ser33/37/Thr41, thereby blocking the classical pro-fibrotic β-catenin signaling pathway. These findings highlight Cdc42 as a promising therapeutic target for kidney fibrosis and suggest DA as an effective Cdc42 inhibitor, offering a novel strategy for the treatment of chronic kidney diseases. Our target identification and validation service employs advanced proteomics and biochemical techniques to discover and confirm therapeutic targets, accelerating the development of innovative treatments for chronic diseases such as kidney fibrosis.

 



Hu, X. et al. Adv Sci (Weinh). 2024.

Figure 3. The Validation of Cdc42  as the Target of Kidney Fibrosis

 

Applications

1. Drug Discovery  

Target identification and validation service provides the foundation for drug discovery by identifying disease-associated molecular targets and confirming their biological functions, accelerating the screening and development of potential drug candidates.  

 

2. Disease Mechanism Research  

By identifying key targets and their signaling pathways, the service enables a deeper understanding of disease mechanisms, uncovering pathogenic factors and supporting the development of personalized treatment strategies.  

 

3. Exploration of Novel Therapeutic Strategies  

Target identification and validation service assesses the role of targets in various physiological and pathological contexts, laying the groundwork for innovative therapies such as targeted treatments, biologics, or PROTAC-based technologies.

 

Deliverables

1. Comprehensive Experimental Details

2. Materials, Instruments, and Methods

3. Relevant Liquid Chromatography and Mass Spectrometry Parameters

4. The Detailed Information of Results

5. Mass Spectrometry Image

6. Raw Data

 

By partnering with MtoZ Biolabs, you gain access to reliable, high-precision target identification and validation service designed to support your research from discovery to application. Let us help you uncover and validate critical therapeutic targets to propel your drug development journey forward.