Adeno Associated Virus Articles & Analysis
7 articles found
In modern biopharmaceutical manufacturing, downstream processing development has become one of the most critical and resource-intensive stages. While upstream cell culture or fermentation focuses on producing the target biomolecule, downstream steps transform crude harvests into highly purified and stable therapeutic products. For biologics such as monoclonal antibodies, recombinant proteins, and ...
In recent years, adeno-associated virus (AAV) particles have emerged as a pivotal tool in the realm of gene therapy, offering researchers and clinicians innovative approaches to treat a variety of genetic disorders. Premade AAV particles represent a significant advancement in this field, streamlining the process of gene delivery and significantly enhancing the efficiency and efficacy of ...
The fast-tracking landscape of contemporary gene therapy demands continuous advances in AAV (Adeno-associated virus) formulation development and the enhancement of cGMP (Current Good Manufacturing Practice) capacity. Together, these transformations are essential in producing safe and effective gene therapies for patients. AAV Formulation Development: A Primary Focus In the field of gene therapy, ...
Recombinase Adeno-Associated Virus (rAAV) particles have taken the center-stage in gene therapy research and development due to their exceptional genetic manipulation capabilities, safety and unprecedented therapeutic success. As biomedical science advances, there is an ever-increasing enthusiasm in the usage of rAAV particles as effective vehicles for gene delivery and the subsequent therapy. ...
In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. Viral vectors have been one of the most widely studied vectors due to their outstanding transduction efficiency and other significant advantages. Viral vector-based gene therapy has now achieved good clinical results. More than a dozen viral gene therapy products have been approved for the treatment ...
What are AAVs? and what makes them good candidates for gene therapy? In two previous posts, we introduced gene therapy, a method for curing genetic diseases by providing healthy copies of defective genes, and Adeno-associated virus (AAV) capsids, the gene therapy delivery system Dyno focuses on. In those posts, we also discussed how natural variants of AAV did not evolve for the specialized ...
A key hurdle to making adeno-associated virus (AAV) capsid mediated gene therapy broadly beneficial to all patients is overcoming pre-existing and therapy-induced immune responses to these vectors. Recent advances in high-throughput DNA synthesis, multiplexing and sequencing technologies have accelerated engineering of improved capsid properties such as production yield, packaging efficiency, ...