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DNA-based Therapy Development Platform

DNA-based therapy development is an emerging therapeutic strategy utilizing DNA molecules and related technologies to treat disease. With a research team with extensive expertise in gene therapy as well as an advanced technology platform, our company is confident to provide customers with DNA-based therapy development services for rare diseases. We offer tailored solutions and analysis workflows to address your specific scientific research requirements, enabling real-time project tracking to expedite your scientific breakthroughs.

Overview of DNA-based Therapy

DNA, one of the most important sources for understanding the fundamental foundations of human life, can be developed as new therapies for disease. Diseases can be treated by replacing defective DNA with normal, healthy DNA. Designing drugs based on DNA sequence and structure can reduce toxicity and side effects. However, DNA-based therapies have poor cellular uptake and undergo rapid degradation in the body, necessitating the use of delivery systems that promote cellular internalization and maintain their activity. DNA-based therapies include DNA aptamers, DNAzymes, plasmids, and oligonucleotides for antisense and antigene applications.

2-1-3-1 DNA-based Therapy Development Platform-1Fig.1 Therapy of DNA modification using CRISPR/Cas9. (Guo, Ning, et al., 2022)

DNA-based Therapy for Rare Diseases

Although most DNA-based therapy is still in the early stages, the emergence of the strategy for treating a variety of rare diseases provides a broad prospect and can help fight cancer, acquired immune deficiency syndrome, neurodegenerative diseases, hemophilia, and other rare diseases. Advances in human genomics, transcriptomics, and proteomics are also further driving the development of DNA-based therapy.

Table 1 Approved DNA-based rare disease gene therapy product. (Arabi, F., et al., 2022)

Cargo Transferred gene/ genetic modification Indication Ex-vivo/in-vivo (target cell) Approving country/Agency Details
DNA Anti-sense oligonucleotide against exon 53 of dystrophin pre-mRNA Duchenne Muscular Dystrophy In-vivo FDA, Japan First DNA-based approved gene therapy product

Our Services

With rich experience in rare disease gene therapy development and a top technical team, our company provides different DNA-based therapy strategies, using different DNA molecules as therapeutic means to achieve DNA editing and repair. We provide DNA vector construction and vector evaluation services to improve gene delivery systems and accelerate your rare disease therapy development.

Plasmid-DNA Therapy Development

Based on advanced technology and a professional team, our company provides customers with plasmid-DNA development and delivery services, through DNA editing and modification to treat related rare diseases.

Single-stranded Circular DNA Therapy Development

Providing the development of single-stranded circular DNA, including production, testing and delivery services, for targeted drug delivery, biomarker analysis, gene editing, and delivery material development.

Non-coding DNA Therapy Development

Our company provides one-stop non-coding DNA therapy development services for in-depth study of various non-coding sequences and mutations.

Why Choose Us?

As an integrated CRO, our company has extensive expertise in DNA-based therapy development for rare disease research. Whatever your specific requirements, we will work closely with you to develop a customized solution. We can quickly respond to the changing needs of your rare disease research projects. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Guo, Ning, et al. "The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy." Journal of Advanced Research 40 (2022): 135-152.
  • Arabi, F., et al. "Gene therapy clinical trials, where do we go? An overview." Biomedicine & Pharmacotherapy 153 (2022): 113324.

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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