Why Choose Recombinant Adeno-Associated Virus Vectors (rAAV) to Treat Eye Diseases

Inherited retinal dystrophies and optic neuropathies cause chronic disabling loss of visual function. With the development of ocular gene therapy, recombinant adeno-associated viral vector (rAAV) is one of the most promising gene enhancement tools for the treatment of inherited eye diseases. Currently, more than 33 rAAV gene therapies are in clinical trials. The current strategy for many ocular gene therapy trials is to shuttle and integrate wild-type copies of the gene into RPE or photoreceptor cells. This is mainly due to the numerous advantages of rAAV DNA vector systems, including: (1) They express transgenes within days or weeks and can reach full expression levels after 4-6 weeks in vivo. (2) The rAAV DNA vector system allows long-term therapy in dogs for at least ten years. (3) The rAAV DNA vector system spreads well within tissues to target large retinal plaques. (4) rAAV injection is very safe, and the number of serious adverse events (SAEs) in clinical trials is very low.

Fig. 1. The development of recombinant AAV vectors targeting ocular diseases. (Buck TM, et al., 2020)

Service Overview

Recombinant adeno-associated virus has been widely used as a delivery system for gene therapy of ocular diseases. As a CRO in the field of ophthalmology, Ace Therapeutics provides global customers with one-stop development services for recombinant adeno-associated virus-based products with high therapeutic potential. Due to the complexity of rAAV treatment and development, our expert team has developed specialized assay kits for the detection and quality control of AAV particle and capsid protein combinations.

• rAAV packaging and purification.
• The process of rAAV infecting cells.
• Analysis and detection.
• Reverse-phase high performance liquid chromatography.
• Anion exchange high performance liquid phase color matching.
• ELISA method.
• Kryptonyl acid analysis.
• N-terminal sequencing.

Explore Our Capabilities

rAAV vector design usually includes capsid design and genome design. AAV capsids determine their unique tissue tropism and host immunogenicity, and capsid modification endows rAAV vectors with new properties and advantages to meet different application requirements. The AAV genome basically contains ITRs-flanked gene expression cassettes, and the modification or design of the genome can change the reproduction ability, infection ability and integration characteristics of the rAAV vector. Correctly designing the transgene cassette and modifying the capsid is critical to generate ideal rAAV vectors for both basic research and clinical applications. Ace Therapeutics scientists offer a variety of custom rAAV vector design and optimization services, including but not limited to:

• rAAV vector design for gene therapy of ocular disease.
• rAAV vector design for basic research of ocular disease.
• Tissue/cell specific targeting rAAV vector service.
• Self-complementary rAAV vector service.

Our Areas of Expertise

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

Ace Therapeutics provides advanced design and modification services for rAAV vector engineering to meet the different needs of gene delivery, gene therapy, basic research and clinical applications. 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. Buck TM, Wijnholds J. Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays. Int J Mol Sci. 2020 12;21(12):4197.

• AAV Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Lentiviral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Non-Viral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Delivery System Development Services for Ocular Nucleic Acid-Based Therapy
• CRISPR/Cas9 Gene Editing Services for Ocular Nucleic Acid-Based Therapy
• Safety and Toxicology Assessment Services for Ocular Nucleic Acid-Based Therapy
• Potency Testing Services for Ocular Nucleic Acid-Based Therapy

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