Advanced AAV Gene Therapy Characterization Technique
Gene therapy is an emerging technology that replaces a faulty gene or adds a new gene to cure disease or improve your body's ability to fight infection. These next-generation treatments hold promise for treating various genetic and chronic conditions, including but not limited to cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS.
Adeno-associated virus (AAV) vectors are the leading form of viral vectors for gene therapy that can potentially treat multiple rare diseases. It utilizes a non-enveloped virus that can be engineered to deliver DNA to target cells to treat diseases. However, one of the primary challenges of large-scale AAV production is the quantification and quality control to determine if the AAV capsid is fully loaded, partially loaded or empty of genetic material.
This technology describes using Charge-Detection Mass Spectrometry (CDMS) and a surface-induced disassociation (SID) device for determining the ratio of empty, partially, and fully loaded AAVs. The inventors have prototyped the method and demonstrated that it could provide highly detailed structural characterization and analysis of AAVs. Additionally, the analyzed AAVs are stable with minimal fragmentation.
This invention can be used to develop AAV gene therapy and evaluate their quantity and quality of AAV loading through the development and manufacturing process.
Existing AAV evaluation techniques only provide information regarding empty and/or filled capsids. This invention enables biotechnology companies and researchers the ability to evaluate full, empty, and partially filled capsids. It could potentially enable the rapid scale-up manufacture of AAV-based therapies.