Toward the industrialization of gene therapies

In response to the Ile de France Region Call for Projects to support novative R & D projects in the field of Bioproduction, POLYTHERAGENE has received financial support for its project: Very efficient transfectant for bioproduction of Recombinant AAV and Lentiviruses for Gene Therapy.

Year after year the successes of gene therapy have been prodigious so that many patients will be able to be treated in the near future. But today, treatments are still far too expensive, mainly because the industrial scale bioproduction of viral particles to introduce genes into diseased cells is difficult and very expensive. Polytheragene, which has know-how in the synthesis of polymers to introduce genes into cells, proposes to develop new molecules and original formulations (polyplexes) with the objective of greatly reducing the quantities of very expensive genetic material used to bioproduce viral particles. These innovative solutions will contribute to the industrialization of gene therapy drugs at more reasonable prices.

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Following a collaboration between Polytheragene, Genethon and Yposkesi (an industrial platform created by the AFM-Telethon in Genopole), the PTG1-plus polymer of the PTG1 family has shown a remarkable transfection efficiency of cells producing viral vectors (AAV and Lentivirus) with remarkably low toxicity.

See the article in Genopole newsletter of April.

A protocol has been developed for the bioproduction of these viral vectors on an industrial scale in bioreactors of 200 to 400 liters. The production of viruses with PTG1-plus was much more effective than that obtained with competing transfection reagents, particularly with respect to the high level of complete viral particles.This scaling up is an essential lever to hope to reduce the costs of production and achieve more reasonable drug prices.

The results were presented at the annual conference of the European Society of Genetic and Cell Therapy (ESGCT) held in Barcelona from 22 to 25 October 2019. Poster P063 and P082

Following these results, Genethon has entered into a license agreement for the use of PTG1 plus. Yposkesi, the leader in Europe for the large-scale production of viral vectors for gene therapy, has also concluded a contract to supply PTG1 plus.

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