Novel AAV Capsids
AAV capsids play a critical role in advancing AAV-based gene therapy. Traditional AAV capsids often suffer from restricted tissue tropism, limited transduction efficiency, and interference from pre-existing neutralizing antibodies, all of which can compromise therapeutic efficacy and safety. Engineered and next-generation AAV capsids enable improved tissue targeting, enhanced gene delivery efficiency, and reduced immunogenicity, allowing lower therapeutic doses and minimizing off-target effects. In particular, novel capsids designed to cross biological barriers—such as the blood–brain barrier—or selectively target specific cell types significantly expand the range of diseases that can be addressed by gene therapy. Furthermore, innovative capsid development supports dose reduction, repeat dosing strategies, and translation from animal models to humans, especially when validated in clinically relevant models such as non-human primates. As gene therapy continues to mature, novel AAV capsids are becoming essential for improving clinical outcomes, enabling precision medicine, and reducing manufacturing and development costs.
AAVnerGene’s AAV Capsid Biodistribution Data
AAVnerGene has generated a comprehensive non-human primate (NHP) biodistribution dataset to evaluate the in vivo performance of both conventional and engineered AAV capsids. Leveraging the ATHENA-I platform, which includes hundreds of distinct AAV variants, these studies assess tissue tropism, transduction efficiency, and specificity across key organs following systemic delivery. please contact us if you when to explore those data.
AAVnerGene's Novel AAV Capsids
AAVnerGene has developed a diverse portfolio of novel and engineered AAV capsids designed to overcome the key limitations of conventional AAV vectors, including suboptimal tissue targeting, limited transduction efficiency, and challenges associated with pre-existing immunity. Our capsids are created through an integrated approach that combines rational design, directed evolution, and high-throughput in vitro and in vivo screening, enabling precise optimization for specific cell types, tissues, and delivery routes. Each candidate capsid is rigorously validated using AAV barcode and next-generation sequencing (NGS)–based technologies to ensure robust performance, reproducibility, and scalability. These efforts are powered by our proprietary ATHENA platform, which comprises three complementary sub-platforms—ATHENA-I, ATHENA-II, and ATHENA-III. By integrating data from all three sub-platforms with artificial intelligence–driven analysis, ATHENA enables efficient identification, evolution, and creation of best-in-class AAV capsids tailored to specific applications. This integrated strategy accelerates development, enhances therapeutic effectiveness, and has the potential to significantly reduce gene therapy development and manufacturing costs.