Généthon and Polyplus-transfection team up to contribute to researching gene therapies for Duchenne Muscular Dystrophy (Article in Pharmaceutical Manufacturing)

Polyplus-transfection SA has become a biotechnology company developing and selling innovative solutions for the delivery of nucleic acids in research, bio-production and therapeutics. Polyplus’ core competence is built around the design, synthesis, characterization and qualification of bio-polymers for highly scalable transient transfection applications as well as the chemical modification of nucleic acids with the goal of enhanced and targeted delivery into living cells.

Généthon, a non-profit biotherapy R&D organization based near Paris, with a mission to design gene therapy products for rare diseases, ensuring their pre-clinical and clinical development and production in order to provide patients with access to these innovative treatments. Généthon, as an integrated R&D center, ensures translational development from research to clinical validation, including bio-manufacturing.

By the mid 2000’s Généthon and Polyplus were working closely together. This was expanded further when Généthon’s search for suitable compliant products to support large scale transient transfection system using suspension cells for the production of viral vectors for its DMD and WAS clinical programs matched Polyplus’ increasing investment in the production of highly characterized and qualified PEI based transfection reagents for use in both pre-clinical and human clinical studies.

Frederic Revah (CEO, Généthon) and Mark Bloomfield (CEO, Polyplus-transfection) recognized there was need for a closer collaboration to secure the development and testing of a PEI-mediated, transient transfection system that could deliver the reproducibility, operator independence and high viral titers needed for Généthon’s DMD clinical program. The outcome of the joint evaluation and development program was the definition and production by Polyplus of a PEI based transfection reagent formulated from a custom synthesized and fully chemically characterized PEI polymer. After formulation in sterile water, the reagent was then extensively qualified using a series of microbiological tests and other tests from the pharmacopeia.

In the hands of the expert teams at Généthon and in Généthon’s proprietary viral production systems, this “highly qualified” reagent, reproducibly delivered high viral titers, independent of the operator and with significantly lower production costs than other transfection technologies.

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