Advantages of Non-Viral Delivery Systems

Nonviral methods for ocular delivery offer several advantages over viral vectors, including a potentially more favorable safety profile due to a lower risk of immunogenicity or insertional mutagenesis, and the ability to deliver large DNA fragments. Viral vectors have been able to attenuate inherited retinal degeneration in mice, but they may be limited by cell tropism, the size of the expression cassette to be transferred, and host immunity to superinfection. More importantly, there is concern that the use of viral vectors in human patients can lead to tumorigenesis and even death. Although newer viral vectors, such as non-integrating and self-suppressing lentiviral vectors, offer promising ways to alleviate some of these concerns, There remains a need to continue to improve and develop gene therapy vectors for use in the eye. Therefore, a large amount of research effort has recently been directed towards the development of non-viral DNA delivery systems, and lipid-based and polymer-based vectors are the main directions for the development of non-viral vectors.

Service Overview

Gene therapy products can utilize non-viral vectors to deliver their genetic material. They generally exhibit lower immunogenicity than viral vectors and are easier to produce on a large scale. Non-viral vectors are usually customized for each genetic payload and application. Therefore, obtaining a stable drug product requires dedicated development coupled with in-depth analytical characterization by scientific experts. Ace Therapeutics has a strong scientific background in the analysis of non-viral vectors and has a broad assay portfolio suitable for the task. We develop the following advanced non-viral vectors for gene therapy of ocular diseases for global customers, including but not limited to.

• Lipid-based NPs
• Peptide-based NPs
• Polymer-based NPs
• Naked DNA

It is worth mentioning that the size and concentration of non-viral vectors are critical to their function of carrying and releasing genetic payloads. Ace Therapeutics has a strong scientific background in the analysis of non-viral vectors, with or without prior separation of the vector from its payload. Our analytical product portfolio spans six orders of magnitude from the low nanometer to the visible size range. This allowed us to accurately determine the absence of free nucleotides and the (sub)visible particle content.

Our Areas of Expertise

• Retinal disease.
• Age-related macular degeneration.
• Hereditary eye disease.
• Glaucoma.
• Corneal disease.
• Optic nerve disease.

The success of gene therapy depends on the creation of safe and efficient gene delivery vehicles. Ace Therapeutics' talented scientists design and construct non-viral vectors for ophthalmic product development for customers worldwide. If you are interested in our services or need more detailed information, please feel free to contact us. Our experienced scientists are ready to help you!

Reference

1. Conley SM, Cai X, Naash MI. Nonviral ocular gene therapy: assessment and future directions. Curr Opin Mol Ther. 2008 ;10(5):456-63. PMID: 18830921; PMCID: PMC2938038.

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