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Volume 2017 eBook, Issue 2
A case study demonstrates that affinity chromatography can offer efficiency and scalability for gene therapy manufacturing using viral vectors.
On Nov. 2, 2012, UniQure’s Glybera, an AAV-1 based drug, was approved in Europe for use in adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD). Since then, interest in gene therapies has skyrocketed, with a fourfold increase in the number of gene therapies being developed between 2012 and 2015. With the power to overcome genetic disorders, these therapies could prove revolutionary to the field of medicine.
The technology aims to treat diseases by delivering genetic material encoding a protein with a therapeutic effect into a patient’s cells. In most gene-therapy studies, a carrier molecule or a vector must be used to deliver the therapeutic gene to the target cells. There are a number of molecular vectors and techniques available for use; however, most frequently this is achieved with viruses.
eBook: Development Strategies for Emerging Therapies
When referring to this article, please cite it as O. Terova et al., "Affinity Chromatography Accelerates Viral
Vector Purification for Gene Therapies," BioPharm International Development Strategies for Emerging Therapies eBook (September 2017).