End-To-End Gene Therapy Solutions
Setting the Standard in Gene Therapy, Together
Gene therapies have potential to transform the way we treat conditions like hereditary genetic disorders, cancer, and infectious diseases, and have become one of the most rapidly evolving fields in regenerative medicine. Delivering them requires optimizing processes upstream and downstream to retain critical quality attributes.
To meet the growing demand for viral vectors in clinical therapeutics, scientists need solutions that offer reproducible performance and scalability with a simplified path to regulatory approvals.
End-to-End Gene Therapy Solutions
Setting the Standard in Reproducible Performance
Boost your gene therapy process performance with transfection reagents and media optimized for specific viral vectors
Ensure a high-performing process with automated, high-throughput, and innovative technologies that speed up development timelines
Enhance process understanding and speed up decision-making with intuitive data analytics
Setting the Standard in Simplified Scale-Up
Simplify the transition to clinical and commercial production with scalable bioreactors, media, and growth factors available in a variety of formats
Streamline revalidation and optimization with scalable purification platform solutions that raise yield and reduce costs
Protect your gene therapy product’s performance during shipping and handling with preassembled, scalable storage solutions.
Setting the Standard for a Simplified Path to Regulatory Compliance
Meet efficacy and safety standards with solutions backed by rigorous quality-assurance processes
Navigate regulatory requirements with our robust analytical tools, testing and validation services, and expert support
Protect against contamination with raw materials, media and buffer filters, and single-use solutions suitable for GMP processes.
Process Development and Manufacturing
End-to-End Solutions for Your Workflow
Browse solutions for gene therapy process development and manufacturing.
Find the Right Solution for Your Process
More About Gene Therapy From Sartorius
Products
Gene Therapy Solutions
Find solutions that accelerate your time-to-market and improve cost-efficiency at every step of your gene therapy workflow.
Request Your Sample | Raw Materials and Consumables
To help find the right materials and consumables for your needs, request a sample from our portfolio.
FAQs - Gene Therapy
Gene therapy is a medical technique designed to correct, suppress, or insert genes within cells to treat or prevent diseases, such as cancer and genetic disorders. The typical delivery methods include using viral vectors, like adeno-associated viruses, or non-viral methods, such as lipid nanoparticles.
Gene therapy can be performed either in vivo or ex vivo. Ex vivo therapies modify cells outside of the body before reintroducing them, which is also referred to as gene-modified cell therapy.
AAV is a small virus less than 30 nm in diameter, containing 5 kb DNA in a capsid with mostly episomal expression. It has different serotypes depending on the capsid's protein expression, supporting targeting of an organ. Lentivirus is more than 100 nm in diameter, and has an envelope surrounding the capsid that contains 9.8 kb RNA. Its integration enables expression in dividing and nondividing cells. Scientists mostly use lentivirus for ex vivo or gene-modified cell therapy.
To treat widely spread diseases like Duchenne muscular dystrophy, demand for viral vector quantity has been increasing, especially for systemic delivery. New strategies have been developed to meet this demand. Improved control of transfection has enabled larger batches, and perfusion systems have supported cell line stability and process intensification. Improved downstream processing technologies have also raised recovery rates.
We're focused on overcoming your challenges in viral vector production. As viral vectors can't go through a viral clearance step, the process needs to mitigate contamination risk. To support this, we provide controlled raw materials, viral media filtration, closed and single-use systems from bioreactors to sterile filtration, and connection and disconnection systems. We also provide fast and high-throughput analytics for process development, manufacturing, and quality control.