Gene Therapy
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.
Key Features & Benefits:
- VGXI brings over 20 years of expertise in high-quality plasmid DNA manufacturing, supporting AAV gene therapy developers from Phase 1 through commercial production, backed by a leadership team with over 150 years of combined experience.
- With a continuously expanding facility in Texas and the largest dedicated footprint globally, VGXI offers exceptional flexibility and the industry’s shortest lead times, enabling program bookings in as little as three months and streamlined MSA execution in approximately 30 days.
- Welcoming all complex plasmids, VGXI’s in-house experts ensure on-time, in-full (OTIF) delivery through advanced facilities and proprietary methodologies, effectively addressing challenges like ITR sequences and orientation variability.
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.
