Bioproduction: how to transition from the experimental phase to the production of treatments for patients

To lead clinical trials and bring products to patients, it is crucial to industrialize the production of innovative treatments to provide clinicians with viral vectors in quantity and quality. Bioproduction refers to all technical processes used for industrial production of biological molecules (including viral vectors).

Genethon’s bioprocesses development team designs, scales and characterizes the production procedures for viral vectors. These methods are then transferred to our exclusive partner for pharmaceutical grade production, Yposkesi, who produces the batches used in Genethon’s clinical trials.

The team also has a role in developing technological innovations to enhance the performance and reliability of vector production processes, both in terms of productivity (increased yield and reduced costs) and of quality of products manufactured.
Around 35 employees are working on the development and improvement of cellular culture and transfection processes (upstream process) and on purification, concentration and formulation (downstream process) of viral vectors. The challenge is to facilitate and secure the transition from laboratory scale to industrial scale, which is particularly complex.

Bioproduction: cell culture and purification

  • Vectors (AAV or lentiviral) are today produced by cells in suspension, in environments without animal-derived components, which helps control the changes of scale needed during development and produce reliably on a large scale. The production processes are developed initially in bottles that are shaken, then transferred to bioreactors whose size is scalable, to ultimately allow their production on an industrial scale (bioreactors of 2L to 200L).
  • Once the vectors are produced by the culture cells, the downstream process enables them to be harvested, then purified to eliminate all impurities and contaminants from cells and/or from the process used, then concentrated and formulated.

Bioproduction: a great many technological innovations

Over the past 15 years, the bioprocesses group has designed and implemented a great many improvements in production methods, but also product analysis that have increased both productivity and quality.

Bioproduction: many applications for treating rare diseases

Over the course of the last several years, Genethon has developed around fifteen vector production processes, as part of therapeutic projects, successfully accompanying their technological transfer to pharmaceutical production.

All of these innovations now make it possible to produce treatments for clinical trials that require large quantities of vectors, and in particular neuromuscular diseases such as Duchenne muscular dystrophy.

Pharmaceutical production: Yposkesi

A pioneer in bioproduction, Genethon set up its own pharmaceutical grade bioproduction facility in 2008. Building on that experience, Genethon, together with AFM-Telethon, Istem, and BPI France, created Yposkesi in 2016. Yposkesi is a European CDMO (Contract Development and Manufacturing Organization) leader in the pharmaceutical production (BPF) of viral vectors for gene therapy. In March 2021, the company increased its production capacity by having the SK group acquire a stake in it, a strategy designed to meet growing global demand for the production of vectors for gene therapy treatments.

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