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Gene Therapy Vector Development Platforms

In gene therapy, the expression of exogenous genetic material in human cells must enter the cell nucleus, which must be achieved through a vector. Based on extensive expertise in viral structure-function relationships, our company is dedicated to providing customers with gene therapy vector development services to generate vectors with novel gene delivery capabilities, thus meeting multiple gene delivery needs in treating rare diseases.

Overview of Gene Therapy Vectors

Currently, gene therapy vectors are generally divided into viral vectors (mainly including lentivirus, adenovirus, retrovirus, adeno-associated virus, etc.) and non-viral vectors (mainly including naked DNA, liposomes, nanocarriers, etc.). Protein engineering technologies have been used in virus vector engineering to effectively deliver exogenous genes, which is expected to expand the utility of engineering vectors. These approaches have shown great promise in vitro, and future in vivo analysis, especially for targeting approaches and solving concerns about the immunogenicity of vectors.

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Fig.1 Viral vectors are used for disease therapeutics. (Dogbey, D. M., et al., 2023)

Gene Therapy Vectors for Rare Diseases

The failure of target genes to be expressed effectively in target cells is currently the greatest difficulty in gene therapy research, and improving the targeting of viral vectors is one way to address this problem. The complexity of viruses, such as complex multimeric protein shells or capsids, presents a challenge in designing viral vectors with new properties, including resistance to immune responses and enhanced targeting ability. The rational design of targeted viral gene delivery vectors for gene therapy has progressed to some extent with success. There are already some cases where vectors have been used in gene therapy for rare diseases:

Table 1 Research of vectors in rare diseases. (Li, H., et al., 2020)

Disease applications Target Edited cells Delivery Sample size Phase Trial number
Mucopolysaccharidosis I IDS gene Hepatocytes AAV 9 I/II NCT03041324
Mucopolysaccharidosis II IDUA gene Hepatocytes AAV 3 I/II NCT02702115
Hemophilia B Factor IX gene Hepatocytes AAV 12 I NCT02695160
Relapsed refractory multiple myeloma, melanoma, synovial sarcoma, and myxoid/round cell liposarcoma NY-ESO-1, TRAC PDCD1 T cells Lentiviral and electroporation 18 I NCT03399448
Relapsed or refractory CD19+ lymphoma TRAC, B2M CAR T cells Lentiviral and electroporation 80 I/II NCT03166878
HIV-1 infection with ALL CCR5 CD34+ HSPCs Liposome and electroporation 5 I NCT03164135
Mesothelin-positive multiple solid tumors PDCD1 and TRAC CAR T cells Lentiviral DNA 10 I NCT03545815
Mesothelin-positive multiple solid tumors PDCD1 and TRAC CAR T cells Lentiviral DNA 10 I NCT03747965

Our Services

With extensive experience in viral and non-viral vector development and a variety of technical capabilities for targeting viral vectors to specific cells, our company provides customers with a wide range of rare disease gene therapy vector development services, designing and producing gene therapy vectors with customized, user-defined gene delivery characteristics. In addition, we provide cell and animal model development services for preclinical evaluation of vector targeting capabilities. Services we can provide include but are not limited to:

Project Workflow

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Equipped with a dedicated research team and advanced technology, our company is committed to providing customers with the genetic incorporation of targeting ligands to produce gene therapy vectors with the desired target specificity. Our scientists specializing in gene therapy vector development will work with you to develop the most appropriate strategy and provide you with the most meaningful data for the development of gene therapies for rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Dogbey, D. M., et al. "Technological advances in the use of viral and non-viral vectors for delivering genetic and non-genetic cargos for cancer therapy." Drug Delivery and Translational Research (2023): 1-20.
  • Li, H., et al. "Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects." Signal transduction and targeted therapy 5.1 (2020): 1.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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