Gene Therapy Research Solutions

Gene Therapy Research Solutions

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The field of gene therapy research is growing with unprecedented speed and influence. In recent years, numerous gene therapy drugs have been approved to market worldwide, this includes many gene therapies developed for different indications which are in the clinical research stage. Gene therapy has brought the first hope to many patients who have sought therapies for years.
As a comprehensive solution provider, Cyagen has established an innovative CRO platform that provides researchers with leading-edge genetically modified model services. We have accumulated a large amount of data on biological information and gene editing over the past decade in our continuous dedication to cell and animal models which have allowed us to play a leading role in the gene-editing field.
By utilizing Cyagen’s in-depth exploration in artificial intelligence (AI), we can provide scientists with advanced gene function analysis and one-stop gene therapy comprehensive solutions, including: target screening and function research, animal model construction, design and packaging of viral vectors such as AAV, LV and ADV, as well as phenotype analysis.
Cyagen provides one-stop solutions for gene therapy development to meet the needs for all levels of research. Our services include CRISPR Cas Screening & Target Validation, Custom Mouse Model Generation, Efficient Adenovirus/Lentivirus Packaging of adeno-associated virus (AAV), lentivirus (LV), adenovirus (ADV), Pharmacology and Pharmacodynamics (PD) Studies, and more. We hope that our services can improve the efficiency of gene therapy research results.


Compared with traditional drug R&D CROs that provide a limited range of services, Cyagen can provide researchers with a more convenient and efficient one-stop service that can effectively help control R&D costs, shorten the development cycle of new drugs, and reduce R&D risks.

Cyagen is a leading life science research model service provider who has developed various robust platforms related to gene therapy covering a variety of rare human diseases.


Gene Prediction and Verification Platform

Viral Vector Design and Development

Custom Animal Model Generation Services

Effectiveness Evaluation

  • AI analysis: Genotype-to-phenotype prediction

  • Construction of target verification model

  • Functional testing


  • Rapid Screening for AAV Serotypes

  • AAV vector optimization

  • AAV packaging (research-grade)

  • Optimized packaging of LV and ADV vectors
  • In vitro validation of Viral Vector


  • Rare disease mouse/rat models

  • Nervous system disease mouse/rat models

  • Drug induction and surgical model

  • Tumor immune mouse/rat models

  • Metabolic disease mouse/rat models

  • Custom mouse/rat model

  • In vivo injection of AAV vector

  • Physiological and pathological testing

  • Molecular testing

  • Behavioral testing
  • Other specific disease phenotype testing

Why Choose Cyagen?


1. Full Service Gene Therapy CRO Platform

Cyagen’s platform covers the entire gene therapy process: from the construction and development of virus vectors, virus packaging and production, creation of gene therapy disease model, to the effective evaluation of gene therapy — our gene therapy CRO platform has rigorous process standards to ensure the consistency and reliability of your research data under a uniformly controlled system.

2. Advanced Bioinformatics Platform

To maximize the impact of Cyagen’s accumulated data on biological information and gene editing, our talented team of big data, AI technology, and bioinformatics background experts is working on establishing a database for gene therapy researchers. This platform uses data sets and algorithm models in the field of gene therapy that will contribute to the risk prediction of human gene mutations and the design of corresponding mouse disease models. Additionally, the database will provide design optimization of gene therapy vector AAV virus capsid proteins., which can be conducive to categorizing and observing disease types, affected protein structure, tissue type, mutation location, and so forth.

3. Leading AI technology supports the optimization and transformation of AAV

The training of AI technology based on deep learning has led to breakthroughs in the optimization and transformation of AAV: quickly and accurately predicts the sequence of AAV capsid protein, obtains the most expected new AAV serotype, and improves upon the effectiveness of AAV vectors and targeted tissue specificity in gene therapy applications. Cyagen’s synthetic biology platform can provide optimized AAV vectors for the development of gene therapy models alongside the necessary downstream validation testing.

4. Risk prediction of pathogenic mutation site

By combining AI technology with our accumulated biological information and gene editing data, Cyagen has established a prediction model for the pathogenicity of gene mutation sites. We can use this prediction model to obtain the mutation sites that are most likely to lead to disease phenotype and construct relevant cell or animal models for verification, which makes a great contribution to the development of gene therapy research.

5. In vivo and in vitro models: one-stop service

In addition to leading the way in custom genetically engineered animal models, Cyagen has also been committed to the field of cell biology for 15 years. We can provide gene editing animal models and virus packaging services to verify gene therapy research. Our products and services have been cited in over 5,700 publications, including high-impact journals such as Nature, Cell, Science, and more. With the aim of contributing to scientific research and clinical transformation of gene therapy, Cyagen has dedicated our experts and resources to support gene therapy research. Cyagen is capable of providing comprehensive services from vector design through research model validation, including pathogenic risk assessment of mutant genes, production of in vitro (cell) and in vivo (mouse/rat) models, phenotypic analysis, gene therapy scheme, and more.
Gene Therapy Case Study

The research groups of Dr. Bin Zhou and the team of Dr. Hefeng Huang co-published an article titled "In Vivo AAV-CRISPR-Pro-mediated Gene Editing Ameliorates Atherosclerosis in Familial Hypercholesterolemia" in the journal Circulation. In this study, researchers find that adeno-associated virus (AAV) delivers CRISPR-Pro to achieve low-density lipoprotein receptor (LDLR) gene correction that can partially rescue LDLR expression and effectively ameliorate atherosclerosis phenotypes in Ldlr mutant mice generated by Cyagen. This finding provides a potential therapeutic method for the treatment of familial hypercholesterolemia (FH).



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