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Toward the industrialization of gene therapies

The Île-de-France Administrative Region is a leader in biotechnologies and innovative biotherapies, as was demonstrated in a note recently published by Institut Paris Region with contributions from Genopole.
Laboratory Laboratory

Polytheragene is a Genopole-accredited biotech located within the Lambe laboratory at the University of Évry. The company is developing a polymer that will improve upon the current market standards for the production of viral particles for therapeutics. The polymer will be used by Genethon and Yposkesi to enhance the bioproduction of gene therapy vectors.

Polytheragene’s polymer, PTG1+, has numerous advantages over conventional transfection agents, providing a massive boost for the large-scale production of lentiviral or adeno-associated virus (AAV) gene therapy vectors.

Results obtained in a partnership between Polytheragene and Genethon have moved the latter, an AFM-Téléthon laboratory, to sign a contract with the former for the use of PTG1+ in the production of its gene therapy products for rare diseases.

Large-scale pharmaceutical-grade lots will be produced by Yposkesi, an industrial facility created by AFM-Téléthon and Bpifrance at Genopole. The productivity increase conferred by PTG1+ will also help bring production costs down and thus contribute to AFM-Téléthon’s objective of more reasonable pricing for these innovative therapies.

Hervé Cheradame, Founder, President and CEO of Polytheragene: “These results illustrate the synergies between Genopole’s actors, be they associations, public laboratories or private companies. And it’s only a start.

Polytheragene will remain the sole owner and unique developer of the polymer. The patented technology was the result of research done at the CNRS, the University of Orléans (prof. C. Pichon and Dr. P. Midoux) and the University of Évry’s Lambe laboratory (Professors H. Cheradame and P. Guegan).

Frédéric Revah, CEO of Genethon: “The technological aspects of bioproduction play key roles in ensuring patient access to innovative medicines and national independence in the domain. We are multiplying our efforts to significantly increase bioproduction yield and the Polytheragene product will enlarge our technological arsenal aimed at achieving that goal.

Alain Lamproye, CEO of Yposkesi: “Innovation is essential if we are to rise to the immense challenges encountered in gene therapy bioprocesses. The development of a suspension-compatible production platform is particularly important in this setting, as it enables an upscaling of processes and responds to commercialization needs. Polytheragene’s product is an essential component for this platform and I am pleased by the cooperation between the three entities for the development of the molecule.

The goal: industrialization of gene therapies

The number of clinical trials in gene therapies continues to grow, notably in such fields as rare immune, blood, vision or muscle diseases, and furthermore in the setting of cancer. However, more common diseases in, for example, dermatology, ophthalmology and neurodegenerative diseases have also become of interest for gene therapies.

Bioreactors producing vectors for gene therapies

The global gene therapy market is estimated at $10 billion today and that number could double by 2023. France, a pioneer in gene therapies, must defend both its place in the global market and its autonomy in the domain, to ensure access to these high-tech therapeutics and minimize its dependence on American or Swiss pharmaceutical giants.

Toward those goals, Yposkesi is France’s principle entity for the pharmaceutical production of gene therapy vectors.

Anne Jouvenceau, Acting Director General of Genopole: “The advancement of gene therapy industrialization is a major and strategic issue for Genopole. The partnership between Polytheragene and Genethon is a vivid example of what it can and should look like. Today, the presence of major entities in gene and cell therapies at Genopole places the biocluster as an essential national actor in the construction of an innovative therapies sector.



Pr Hervé Cheradame

Article posted on 26 March 2020


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