Advancing AAV Research With Precision Analytical Tools

- AAV Titration ELISA Kits Adeno-associated virus (AAV) vectors have emerged as a pivotal tool in gene therapy, offering a promising delivery system in treating genetic disorders. These small, non-pathogenic viruses belong to the Parvoviridae family and are characterized by their capsid proteins: VP1, VP2, and VP3. Mutations in these capsid proteins give rise to various AAV serotypes, with over ten well- characterized types (AAV1 to AAV10) exhibiting distinct tissue tropisms and immune profiles. This diversity provides researchers with a versatile toolkit for targeting specific tissues while minimizing immunogenicity. The popularity of AAV vectors in gene therapy stems from their exceptional biosafety profile, stable gene expression, and low immunogenicity. These attributes, coupled with their broad host range and strong tissue specificity, have led to their widespread adoption, with AAV-based delivery systems now accounting for over 70% of gene therapy drugs. The significant market potential and growing demand for effective gene therapies have spurred substantial investment in AAV vector development by numerous biotechnology companies. Recent years have witnessed remarkable progress in AAV-based gene therapies, with 2023 marking a watershed moment for the field. The U.S. Food and Drug Administration (FDA) approved a record- breaking www.acrobiosystems.com Solutions for AAV Research & Development: AAV Titration and Anti-AAV Antibody ELISA Kits Table 1. Current FDA-approved gene therapies E-mail: order@acrobiosystems.com Tel: +1(800)810-0816 Web: www.acrobiosystems.com seven gene therapies that year, setting a new benchmark for the industry. This surge in approvals has catalyzed the growth of the global AAV therapy market, which reached $1.5 billion in 2023 and is projected to expand to $22.3 billion by 2029. Currently, six AAV-based gene therapies have received regulatory approval, with five gaining FDA authorization. The most recent approval in June 2023 underscores the rapid pace of innovation in this dynamic field. As AAV vector applications in gene therapy continue to expand, the need for precise and reliable tools to support research and development becomes increasingly critical. Two fundamental aspects of AAV research are the accurate quantification of AAV capsid titers and the detection of pre-existing antibodies against AAV. These measurements are essential for ensuring the safety, efficacy, and proper dosing of gene therapies, as well as facilitating the successful translation of gene therapies from the laboratory to clinical applications. AAV therapy manufacturing involves complex processes where accurate capsid titration is crucial for quality control. Precise measurements ensure adequate viral capsid production, which is essential Name Luxturna AAV2 RPE65 Leber Congenital Amaurosis May 2017 $125M $126M $850,000(S425,000 per eye; one-time) $631,000 per year ($24,250 per vial) Zynteglo LV HBB Beta Thalassemia August 2022 $2.8M (one-time) $3M $162M Hemgenix AAV5 AAVrh74 HSV-1 AAV5 Elevidys Vyjuvek Roctavian FIX Hemophilia B November 2022 May 2023 June 2023 June 2023 Dystrophic Epidermolysi-s Bullosa Duchenne Muscular Dystrophy(DMD) Hemophilia A COL7A1 DMD FVIII - - - - $67M $2.731M $3.5M(one-time) $3.2M(one-time) Zolgensma AAV9 SMN1 Spinal Muscular Atrophy(SMA) May 2019 $2.1M (one-time) $1.37M $1.985M Skysona LV ABCD1 Cerebral Adrenoleuko-dystrophy September 2022 $1M $41M $636M $1.430M $3M (one-time) $2.9M (one-time) Vehicle Target Gene Disease 2022 Sales (UsD) Projected 2028 Sales (usD) Cost/Patient (USD) FDAApproval - AAV Titer ELISA Assay Kit - AAV Antibody Assay Kit Strong specificity, no cross-reactivity with other AAV serotypes. High sensitivity, detects as low as 1.70E+08 capsids/mL. Wide detection range, from 1.70E+08 capsids/mL to 1.09E+10 capsids/mL. Quick detection, completed in 2 hours and 20 minutes. High accuracy, with sample recovery rates between 80% and 120%. Rigorous methodology validation, complete validation report available. Fully validated with high sensitivity, specificity, and precision, ideal for pre-existing antibody detection in AAV gene therapy clinical trials, facilitating rapid sample screening. www.acrobiosystems.com Advanced Solutions for AAV Gene Therapy Research AAV5 Titration ELISA Kit AAV8 Titration ELISA Kit AAV3 Titration ELISA Kit AAV2/9 Titration ELISA Kit Anti-AAV5 Antibody ELISA Kit Anti-AAV8 Antibody ELISA Kit Anti-AAV9 Antibody ELISA Kit Anti-AAV2 Antibody ELISA Kit The specificity of AAV-A005H (AAV5 Titration ELISA Kit - Fast) was evaluated against AAV1/2/3/8/9/dj serotypes. Results demonstrated exclusive binding of AAV-A005 to AAV5, indicating its specificity. The specific binding of AAV-A005H(AAV5 Titration ELISA Kit-Fast) to native/denatured AAV5 was detected. The results showed that AAV-A005H only binds specifically to native AAV5. (Denatured AAV5: 95℃ for 10min) AAV-A005H (AAV5 Titration ELISA Kit - Fast) can detect AAV5 capsids from various manufacturers with consistent sensitivity. Immobilized AAV8 Capsid Protein can bind Anti-AAV8 Antibody in 1:50 human serum, and then add Biotin-AAV8 Capsid Protein. Detection was performed using Streptavidin-HRP with sensitivity of 46.9 ng/mL. The specific binding of PAV-A008 (Anti-AAV8 Antibody ELISA Kit) to Anti-AAV3/5/9 antibody was detected. The results showed that PAV-A008 only binds specifically to Anti-AAV8 antibody. E-mail: order@acrobiosystems.com Tel: +1(800)810-0816 Web: www.acrobiosystems.com Discover our latest additions for AAV gene therapy research: the AAV Titer ELISA assay kit and AAV Antibody assay kit. Both kits are meticulously validated for high specificity, sensitivity, and accuracy, offering unparalleled performance for your research needs. Learn more Figure 1. Summary of mechanism of action of gene therapy using AAV vector (adapted from https://encyclopedia.pub/entry/37119) Figure 2. Correlation between ELISA and neutralization assays for rhesus macaque samples pre- and post-AAV inoculation Mol Ther Methods Clin Dev. 2022;24:199-206. E-mail: order@acrobiosystems.com Tel: +1(800)810-0816 Web: www.acrobiosystems.com - High-Sensitivity Anti-AAV Antibody ELISA Kits Pre-existing antibodies against AAV vectors present a significant challenge in AAV-mediated gene therapy, potentially compromising treatment efficacy in both human patients and animal models. While cell-based assays have traditionally been used to detect neutralizing antibodies, ELISA-based anti-AAV antibody detection methods have emerged as valuable and convenient tools for rapid initial screening. ELISA-based anti-AAV antibody detection offers several advantages, including simpler operation, faster turnaround, and cost-effectiveness, especially when processing large sample numbers. A comparative study of AAV1, AAV8, and AAV9 serotypes in rhesus monkey and human sera revealed high consistency (Pearson r > 0.8) between ELISA and cell-based assays, validating ELISA as an effective screening method for both preclinical and clinical applications. Anti-AAV antibody ELISA kits provide quantitative, reproducible results crucial for establishing antibody titer thresholds that may impact treatment outcomes. Their serotype-specific detection capabilities aid in selecting appropriate AAV vectors, while their ability to detect both neutralizing and non-neutralizing antibodies offers comprehensive immune response profiling. Although cell-based assays remain the gold standard for detailed characterization, ELISA kits offer a practical and efficient method for initial screening and monitoring. AAV Titration and Anti-AAV Antibody ELISA kits serve as effective analytical tools in advancing AAV-based gene therapies. The AAV Titration ELISA Kit ensures precise quantification of viral capsids, facilitating accurate dosing and stringent quality control in vector production, while the Anti-AAV Antibody ELISA Kit enables efficient screening for pre-existing neutralizing antibodies that could impact treatment efficacy. Together, these assays enhance the robustness of gene therapy development by streamlining patient stratification, optimizing preclinical studies, and generating comprehensive data for regulatory submissions. As the field of AAV-based gene therapy continues to evolve, the integration of these standardized analytical methods not only enhances current research efficiency but also establishes a foundation for future innovations, ultimately accelerating the translation of promising therapeutic candidates from benchtop to clinic. ACROBiosystems is pleased to announce the launch of a new series of AAV Titration ELISA kits and Anti-AAV Antibody ELISA kits, expertly designed to advance AAV gene therapy research. These kits feature exceptional sensitivity, specificity, and accuracy, enabling rapid and precise AAV titration and sample screening. Tailored for rigorous scientific applications, they provide researchers with reliable tools to advance their studies in gene therapy. for therapeutic effectiveness in both preclinical and clinical phases. Consistent titer measurements guide dosing regimens, balancing efficacy and safety. The AAV titration ELISA Kit provides precise quantification of AAV capsids in a biological sample, critical for determining correct therapeutic dosages and maintaining production QC. This kit ensures reliable measurements, optimizing production processes and meeting regulatory standards for clinical applications. Regulatory bodies like the FDA require accurate viral titer measurements to meet identity, potency, quality, and purity standards. The AAV titration ELISA Kit can help support compliance with regulations by offering reproducible tools for measuring AAV capsid concentration. This facilitates regulatory submissions and approvals, advancing the development and commercialization of AAV-based therapies while potentially improving patient outcomes. AAV5 AAV8 AAV3 AAV2 AAV9 AAV5 AAV8 AAV9 AAV2 Titration ELISA Kit (Fast) Antibody ELISA Kit AAV Capsid Titration Detection Antibody Pair (Universal) Explore our products