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Animal Models Development Service

Drug development for rare diseases is faced with the dilemma of a small number of patients, scarcity of research samples, and high difficulty of clinical trials. One of the major problems hindering drug development for rare diseases is the lack of suitable animal models in preclinical studies. The establishment and availability of stable and reliable animal models of rare diseases are irreplaceable for the study of rare diseases. They have an inestimable potential for the study of rare disease pathogenesis, drug target research, and therapeutic effects evaluation. Several excellent models have already been developed, such as the GAA-knockout mouse model for Pompe's disease and the Sgca-null mouse model for α-sarcoglycanopathy. Some of these have already led to the development of much-needed therapies such as ERTs. Nevertheless, there is a continuing need to develop more effective models to facilitate drug development and clinical trial design for rare diseases.

Fig. 1 Animal models presented to the EMA Committee for Orphan Medicinal Products.

Fig. 1 Animal models presented to the EMA Committee for Orphan Medicinal Products. (Vaquer G, et al., 2013)

Our team of experts with extensive expertise in animal model pharmacology and efficacy research is dedicated to developing accurate and reliable animal models to support the advancement of rare diseases and related gene therapy research. With years of accumulated experience and long-term practical trials, we are well-positioned to help our clients develop efficient preclinical disease models in areas such as metabolic, neuromuscular, and ophthalmic rare diseases. These animal models provide a valuable platform for studying rare disease mechanisms, target validation, and drug screening.

Our Services

Our company has established a series of research and development technologies for genetically modified animal models, such as gene cloning, transgenesis, gene knockout plasmid construction, experimental animal embryonic stem cell manipulation, microinjection, genotype identification, strain breeding, and phenotype analysis. If you have any plans for rare disease model development, our expert team is ready to provide you with a complete service process, including protocol customization, animal modeling, and subsequent animal experiments.

Advantages of Our Animal Model Platform

  • No off-target effects, accurate modifications, short cycle time
  • Professional technical support
  • The rich experience accumulated in successful cases of new drug development
  • Timely project reporting and after-sales service

As an excellent provider of preclinical research services, our company offers efficient gene modification animal model customization services, disease animal modeling services, phenotypic analysis services, etc. for scientific research institutes, hospitals, and pharmaceutical companies. The animal models we develop will provide an effective platform for you to study the efficacy of candidate therapies in animal models and to confirm and/or elucidate the anticipated mechanism of action. If you are interested in our services, please contact us for more details.


  • Vaquer, G.; et al. Animal models for metabolic, neuromuscular, and ophthalmological rare diseases. Nature Reviews Drug Discovery, 2013, 12(4): 287-305.

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