Advantages of Adeno-Associated Virus (AAV)

As gene therapy is being explored as a treatment modality for an increasing number of ocular diseases, drug development of adeno-associated virus-based ocular gene therapy products is also rapidly advancing to help establish powerful new therapeutic products. Due to its higher specificity, efficiency and safety, AAV has become the main carrier of ocular gene therapy. It is a small (25 nm) non-enveloped virus of the Parvoviridae family that has many advantages as a delivery system, including favorable retinal cell transduction properties, and because AAV requires a helper virus for replication, it is generally considered safe and has low pathogenicity in humans.

Fig. 1. Schematic representation of AAV genomic structure. (Srivastava A, et al., 2020)

Service Overview

Ace Therapeutics is a comprehensive preclinical CRO for ophthalmology. Our talented scientists have extensive experience in the field of gene therapy. We can customize exclusive gene therapy product development services according to your research project.

Formulation and production of AAV products requires careful selection of conditions to ensure good stability and yield, as some common processing methods used for biologics, such as filtration or lyophilization, can lead to aggregation or loss of AAV titers. Maintaining stability is also a major challenge when choosing storage conditions. Additionally, due to the inherent complexity of AAV products, a range of advanced analytical tools are often required to fully understand the physicochemical properties, purity and potency of the drug substance (DS) and product (DP). With professional experience in the field of nucleic acid-based therapy and AAV development, Ace Therapeutics provides the following services for the development of AAV-based ocular therapeutics, including but not limited to:

• Vector selection and characterization.
• AAV production and purification.
• Formula development.
• Screening of drug delivery system.

What Can We do?

With a deep understanding of the sensitivities and evolving landscape of ocular gene therapy development, we apply the principles of design excellence to provide services related to gene therapy for ocular diseases, always meeting the needs of our customers at every stage of development.

Our ocular-specific gene delivery platform enables AAV design, AAV purification, AAV titration, and AAV toxicity and safety assays to meet specific needs.

We are proud to provide customized AAV services for ocular diseases to meet the needs of basic research and clinical applications.

AAV-based gene therapy research is under development for different ocular genetic diseases, and positive data demonstrate the therapeutic potential of AAV-based gene therapy in ocular disease. Ace Therapeutics has built a team of experienced scientists with extensive hands-on experience supporting gene therapy development efforts for ocular diseases. We will also apply our extensive knowledge to lead the bioanalytical testing of your promising gene therapy products. 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!

References

1. Srivastava A, Mallela KMG, Deorkar N, et al. Manufacturing Challenges and Rational Formulation Development for AAV Viral Vectors. J Pharm Sci. 2021 Jul;110(7):2609-2624.
2. Rodrigues GA, Shalaev E, Karami TK, et al. Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye. Pharm Res. 2018 27;36(2):291.

• Lentiviral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Non-Viral Vector Development Services for Ocular Nucleic Acid-Based Therapy
• Recombinant Adeno-Associated Virus 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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