Background and history of cell lines for biological research and manufacturing
The sequencing of the human genome was declared complete on April 14, 20031, two years earlier than planned, and was a watershed event in biotechnology. Given that the sequencing of the human genome provided the ability to “read” genes of a patient, one might say that the first direct therapeutic benefit would be the creation of a therapy that “wrote” a gene to correct a defect. This concept of “gene therapy” long predated the human genome project, but the project certainly accelerated its progress. The first gene therapy approved in the Western world was Glybera in July 2012. Glybera, using an engineered virus as a vector, delivered the gene for the enzyme lipoprotein lipase to the cells of patients with lipoprotein lipase deficiency, a genetic disorder in which a person has a defective gene for the enzyme. Although it is no longer marketed and was not a commercial success, it led the way to subsequent gene therapies that are treating diseases including cancer, inherited retinal dystrophies, and spinal muscular atrophy. Gene therapy has, in a real way, made “the blind see and the lame walk” (Matthew 11:5).