At Protheragen, our cutting-edge adeno-associated virus (AAV) vector platform is revolutionizing the field of gene therapy. It streamlines the development process of AAV vectors while significantly enhancing their functionality. Our platform has successfully overcome the challenges of translating therapies from animal models to human applications. With this advanced technology, we are proud to showcase our dedication to innovation in gene therapy and vector delivery systems. We are uniquely positioned to offer comprehensive, one-stop therapy development services for a wide range of diseases, leveraging the full potential of our AAV vector platform.
Adeno-associated virus (AAV) vectors have become a cornerstone in the field of gene therapy. Their remarkable ability to deliver genetic material into host cells with high efficiency and low immunogenicity sets them apart. Derived from the naturally occurring AAV, a small, non-enveloped virus within the Parvoviridae family, AAV vectors possess several key characteristics that make them ideal for gene therapy applications:
AAV vectors can be classified according to the serotype of the AAV capsid. Each serotype has unique tissue tropisms and immune profiles, which significantly impact their applicability in specific therapeutic contexts. Here are some commonly used AAV serotypes in gene therapy:
Fig.1 Electrostatic potential of AAV capsids. (Suarez-Amaran, L., et al., 2025)
The field of AAV-based therapy development is rapidly advancing, with numerous clinical trials underway and several therapies already approved by regulatory agencies. The success of AAV-based gene therapies in treating genetic disorders has validated the potential of this approach. However, challenges remain, including the need for more efficient vector delivery systems, the management of immune responses, and the optimization of vector production processes. Ongoing research focuses on developing next-generation AAV vectors with enhanced tissue specificity, reduced immunogenicity, and improved transduction efficiency. Additionally, the scalability and cost-effectiveness of AAV vector production are critical factors for the widespread adoption of these therapies.
Table 1. Approved AAV-based gene therapy products. (Arabi F., et al., 2022)
| Year of Approval | Trade name (General name) | Manufacturer | Vector | Transferred gene/ genetic modification | Indication | Ex-vivo/in-vivo (target cell) | Approving country/Agency | Details |
|---|---|---|---|---|---|---|---|---|
| 2012 | Glybera (alipogene tiparvovec) | UniQure | AAV1 | lipoprotein lipase (LPL S447X) | Familial lipoprotein lipase deficiency | In-vivo | EMA | First gene therapy product in the European Union, later withdrawn from the market in 2017 |
| 2017 | Luxturna (Voretigene Neparvovec-rzyl) | Spark Therapeutics (Roche) | AAV2 | RPE65 | RPE65 mutation-associated retinal dystrophy | In-vivo | FDA, EMA, UK, Australia, Canada, South Korea | First FDA-approved In-vivo AAV gene therapy product |
| 2019 | Zolgensma (Onasemnogene Abeparvovec-xioi) | Novartis | AAV9 | SMN1 | Pediatric Spinal Muscular Atrophy | In-vivo | FDA, EMA, UK, Japan, Australia, Canada, Brazil, Israel, Taiwan, South Korea | Most expensive drug worldwide |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Protheragen’s trio of AAVLinkTM platforms revolutionizes the creation of AAV vectors by simplifying the development process and maximizing their potential. Our proprietary AAV platform is a testament to our relentless pursuit of innovation.
AAV-Find Platform
Unlock the potential of groundbreaking AAV variants that offer superior tissue-specific targeting, driving the future of precision medicine. Our expertise in bridging the gap from animal models to human applications ensures seamless translation, setting the stage for transformative progress in gene therapy and customized therapeutic approaches.
AAV-Stable Platform
Leveraging inducible cell lines, we have revolutionized the manufacturing of AAV vectors, ensuring stable and scalable production. This cutting-edge method guarantees consistent quality and efficiency, allowing us to fulfill the growing demand for AAV vectors in a sustainable and controlled way.
AAV-Expand Platform
Harnessing dual or multiple AAV systems, we surmount packaging limitations to deliver larger therapeutic genes with unparalleled precision and efficacy. This pioneering strategy unlocks the full potential of AAV vectors, transcending size barriers and broadening the horizons of gene therapy applications for intricate genetic conditions.
Protheragen provides a suite of AAV vector design services tailored to the precise needs of gene therapy research and development. Our services are structured to cover every aspect of AAV vector development, from DNA fragment preparation to final delivery.
Protheragen, a leading provider of advanced gene therapy solutions, offers a comprehensive suite of AAV packaging services to support researchers and developers in the field of gene therapy. Our state-of-the-art facilities and experienced team of scientists are dedicated to delivering high-quality, customized AAV vectors tailored to the specific needs of our clients.
At Protheragen, our AAV purification services are distinguished by our commitment to scientific rigor, our use of state-of-the-art methodologies, and our ability to customize our services to meet specific project needs.
Protheragen is committed to staying at the forefront of AAV characterization technology, continuously refining our methods, and integrating new advances to provide the best possible services to our clients.
Protheragen employs a suite of methodologies to construct and utilize AAV capsid libraries, ensuring the generation of high-quality, diverse viral vectors for gene therapy applications.
AAV Vector Design and Optimization
We offer comprehensive support for the design and optimization of rAAV vectors, from selecting optimal promoters and transgene cassettes to incorporating regulatory elements. Our goal is to achieve robust and sustained gene expression, tailored to meet specific therapeutic needs.
AAV Production and Purification
Utilizing our cutting-edge GMP-compliant facilities and proprietary manufacturing technologies, we deliver reliable and scalable AAV production and purification services. Our solutions are designed to meet the diverse requirements of our clients, ensuring high-quality vectors for every application.
AAV Characterization and Quality Control
Our extensive analytical capabilities allow for thorough characterization of AAV products. We assess physical, biochemical, and functional properties to ensure consistent quality and safety, providing confidence for downstream applications.
Preclinical Evaluation and Validation
We perform rigorous in vitro and in vivo studies to evaluate the efficacy, biodistribution, and safety of our AAV-based gene therapy candidates. This comprehensive approach generates valuable data to support the successful translation of our therapies into clinical settings.
References
Our AAVLinkTM platform is at the forefront of adeno-associated viral (AAV) vector development, leveraging our extensive expertise and state-of-the-art technologies to drive innovation in gene therapy.
