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In ex vivo gene therapies, such as CAR-T therapeutics, the patient's own cells (autologous) or healthy donor cells (allogeneic) are modified outside the body (hence, ex vivo) using a vector to express a particular protein, such as a chimeric antigen receptor. [62]
In science, ex vivo refers to experimentation or measurements done in or on tissue from an organism in an external environment with minimal alteration of natural conditions. [2] A primary advantage of using ex vivo tissues is the ability to perform tests or measurements that would otherwise not be possible or ethical in living subjects
The most common form of gene therapy involves inserting a normal gene to replace an abnormal gene. Other approaches include repairing an abnormal gene and altering the degree to which a gene is turned on or off. Two basic methodologies are utilized to transfer vectors into target tissues; Ex vivo gene transfer and In vivo gene transfer.
Two methods of gene therapy using lentiviruses have been proposed. In the ex vivo methodology, cells are extracted from a patient and then cultured. A lentiviral vector carrying therapeutic transgenes are then introduced to the culture to infect them. The now modified cells continue to be cultured until they can be infused into the patient.
The majority of gene therapy approaches leverage viral vectors, such as adeno-associated viruses (AAVs), adenoviruses (AV), and lentiviruses (LV), to facilitate the insertion or replacement of transgenes either in vivo or ex vivo. These vectors serve as delivery vehicles for introducing the therapeutic genetic material into the patient's cells.
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...