A brief guide on designing a comprehensive cell and gene therapy bioanalytical strategy
The field of cell and gene therapy (CGT) is rapidly developing, with the number of therapies in development greatly increasing over the last five years. The application of CGT provides the promise of significant long-term health benefits to people suffering from a wide range of diseases, from ophthalmological disorders to cancer.
In cell therapies, cells from a donor (allogeneic therapies) or from the patient (autologous therapies) are propagated outside the body and then introduced into the patient1. In gene therapies, modifications to the genetic material of somatic cells can be conducted inside (in vivo) or outside (ex vivo) the body1. Functional copies of a mutated gene can be inserted or new proteins related to the therapeutic approach can be introduced to the patient’s genome. For example, an inherited disease may involve a genetic mutation in a gene for a key metabolic enzyme, resulting in a nonfunctional enzyme and toxic accumulation of the enzyme’s substrate in the body. A gene therapy would introduce a functional gene into the body with the use of a viral vector or lipid nanoparticle. Genetic modifications can allow for constitutive production of the replacement enzymes, providing significant advantages over alternative approaches that require regular dosing of exogenous enzyme replacement therapies. Current research is also aiming to develop therapeutics that perform finer-scale editing of genetic mutations inside cells (“gene editing”)1.
- Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M. Gene therapy comes of age. Science (80- ). 2018;359(6372). doi:10.1126/science.aan4672
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