While the first gene therapy drugs are obtaining marketing authorization and being made available to patients, the challenge of industrializing their production is a crucial one.
What is bioproduction?
Bioproduction refers to all technical and manufacturing processes used for industrial production of bio-medications, including gene therapy vectors.
Manufacturing these treatments intended to produce a therapeutic protein in a patient requires a high-caliber technological environment and very precise procedures. These innovative therapies are very complex to produce. They require research and development of technological innovations to considerably improve their yield and reduce their very high cost.
France has spearheaded the first gene therapy successes, and so absolutely must meet this challenge of bioproduction.
How to produce gene therapy treatments?
Gene therapy involves administering a gene drug to the diseased cells using a virus that has been rendered harmless, used as a “vehicle” or vector.
To manufacture these treatments we need cells that serve as vector factories and pieces of DNA: the therapeutic gene and the genes needed to product “vector-viruses”. The production method may be different according to the type of vectors and quantities desired.
Industrializing production: a major challenge
At a time when gene therapies are developing exponentially, nobody is able to produce enough treatments for all patients. In this international competition, it is an opportunity for French research, which is responsible for several world firsts, to further and develop its expertise in gene therapy bioproduction within an industrial sector. And a crucial factor is guaranteeing its health and financial independence.
To meet growing needs, we need to increase not only production capacities, but also yields in order to drastically reduce costs.
“This change of scale requires in-depth knowledge of viruses, the cells that produce these vectors. We are at the frontier of basic research, applied research and industry,” explains Serge Braun, scientific director of AFM-Telethon.
This is why, in the race to change scale, AFM-Telethon and Bpifrance created YposKesi in 2016, an industrial platform devoted entirely to the production of gene therapy drugs. Its goal is to accelerate the technological progress that will yield productivity gains and reduce production costs for these drugs.