Gene Therapy

Gene Therapy Icon

Gene therapy is an innovative approach that involves the introduction or modification of genetic material into a patient’s cells to correct or replace faulty genes, aiming to treat or even cure genetic diseases. Plasmid DNA plays a critical role in the development and production of viral vector-based gene therapies. Viral vectors are modified forms of viruses that have been engineered to deliver therapeutic genetic material to target cells. Some of the most used viral vectors in gene therapy include lentiviruses, adenoviruses, and adeno-associated viruses (AAV). Of these, AAVs have gained significant popularity for gene therapy due to their excellent safety profile, tissue-targeting capabilities, potential for long-term gene expression, low immunogenicity, and proven clinical success.

In the production of adeno-associated virus vectors for gene therapy, several plasmids are transfected into producer cell lines for viral particle production, each playing a specific role in the vector construction process.

VGXI has a demonstrated track record in manufacturing plasmids critical for a range of AAV serotypes across diverse scales and quality tiers – from Pre-Clinical, HD, and cGMP levels. We provide comprehensive support from the very start, from initial plasmid design and master cell bank generation to process development activities to customize and optimize manufacturing based on specific project needs (i.e., with or without antibiotic selection). Trust us to help you to advance your gene therapy programs with success—on budget, and on time.

AAV Transfer Plasmid
AAV Transfer Plasmid contains the therapeutic gene of interest (GOI) under the control of a suitable promoter and regulatory elements.
Replication (Rep) and Integration (Cap) Plasmid
Replication (Rep) and Integration (Cap) Plasmid encode the AAV replication and capsid genes necessary for AAV replication and determines the AAV serotype and tissue tropism.
Helper Plasmid
Helper Plasmid supplies AAV genes not in the Rep and Cap plasmids needed for AAV replication and packaging. These genes are excluded from the final vector to prevent the formation of replication-competent virus.