Target-to-Hit Services

Successful medicine begins with target validation and target discovery. Finding druggable targets remains pivotal to the future R&D success of pharmaceutical companies. WuXi Biology’s innovative Advanced Gene Manipulation (AGM™) technology and platform serve clients in the identification and validation of both known and novel targets to understand their contexts of diseases and accelerate go vs. no-go decisions thus reducing the risks that impact clients’ drug R&D portfolios.

As one of WuXi AppTec’s well recognized strengths, our target validation program has teamed with several major pharmaceutical companies for years to support their programs from oncology, metabolic diseases, translational medicine, to pharmacogenomics. This has resulted in more than 50 various potential drug targets delivered to our clients.


Drug Target Validation

Gene manipulation of in vitro and in vivo

  • Target knockdown, co-knockdown of isoforms, and target overexpression
  • Conventional knockout, knock-in; conditional regional, organ, or cell type specific knockout in mice or isogenic cell lines
  • Region specific or cell type specific inducible/reversible transgenic mice
  • BAC/YAC transgenic mice for humanized animal models
  • Evaluation of target functional roles
  • Characterization of target activities & correlations in cell lines with positive and negative effects
  • Rescue study for target specificity confirmation

Genetic approaches

  • Viral-based or nonviral-based vectors in specific cell, tissue, organ or systemic gene delivery & manipulation in vitro & in vivo
  • Transgenic technology for model generation; genotyping; breeding & characterization
  • Targeted genome editing platform (CRISPR/cas 9) for knockout, knock-in of genome manipulation at cell types of interest

Target Engagement and MOA

Gene target in contexts of disease model

  • Pathway dissecting & phenotypic study
  • In vitro functional readout & evaluation
  • MOA & signaling cascade
  • Targeting specific tissues for MOA study
  • In vivo phenotypic characterizations in various disease models
  • shRNA or sgRNA library screening for target id
  • IHC, FACS, WB, qPCR for target engagement

Translational Predictive Biomarkers

  • Multiple marker classes including DNA, RNA, protein & microRNA markers
  • Genetically defined platform including both in vitro & in vivo disease models
  • Assay development using both mouse & human samples
  • Bioinformatics supports

Gene Manipulation | AAV Vector Services

In vivo gene manipulation services