The pharmaceutical company Lonza has just opened a new state-of-the-art manufacturing facility to produce cell and gene therapies. You can find the full press release here, on Lonza’s website.
The new facility is designed to be a center of excellence for the treatment of rare genetic diseases and life-threatening conditions. It is the largest facility of its kind, spanning 300,000 square feet, or 27,870 square meters. The center has been equipped with state of the art technology and is designed to support the entire process of treatment development, from initial research to pre-clinical and clinical trials, and through to commercialisation. By the end of this year approximately 200 people are expected to work at the center, and this number may increase. Lonza built it in response to, and anticipation of, a growing demand for cell and gene therapies for the treatment of rare and serious illness.
Cell and gene therapies are two relatively new and very promising areas of treatment development. Gene therapies treat diseases that result from genetic mutations by replacing the faulty gene with a functioning version created by researchers. The gene is designed to then work as normal, which should reduce or in some cases even eliminate the symptoms of the disease. There are two forms of gene therapy; in vivo, where the genes are administered to cells inside the patient’s body, and ex vivo, in which cells are taken from a patient, modified, and then returned. In order to merge the gene into the cells a carrier, known as a vector, is used. This is often through a safe virus, but there are also other forms of vector that can be used.
The second type of treatment, cell therapy, involves taking certain types of living cell and introducing them into a patient’s body. This is so that the new cells can grow and replace or repair the patient’s damaged cells that contribute to their disease. The living cells can be taken from either a donor or the patient themselves.