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NEW TECHNOLOGY: Polyplus-transfection develops GMP-grade therapeutic virus reagent

Polyplus 2018 XL 059 lo resTransfection is the way to deliver exogenous nucleic acids such as DNA, RNA or oligonucleotides into cells and these nucleic acids can be transported by polymeric or lipidic transfection reagents that facilitate their cellular uptake. This process is commonly used for genomic studies such as gene expression, screening, RNA interference and in-vivo research but can also be performed for bioproduction (virus and protein production) or therapeutic purposes (human gene therapy).

Delivery of nucleic acids into cells can be achieved by different physical methods such as electroporation, sonoporation or microinjection, but these processes are relatively toxic for the cells: transfection with chemical compounds is a more reliable alternative that maintains significantly higher cell viability.

Dr Jean-Paul Behr, one of the founders of France-based transfection reagents specialist company Polyplus-transfection, is a renowned scientist whose research focus is centred on DNA-binding molecules relevant to biotechnology and gene therapy. Pioneering work from Professor Behr’s laboratory led to the commercialisation of well-known transfection reagents such as Transfectam, Lipofectamine and Polyethylenimine (PEI) and he co-founded Polyplus-transfection in January 2001 to make highly effective and easy-to-use transfection reagents commercially available to research scientists working in molecular and cell biology. There are several other companies working on transfection: ThermoFisher, MirusBio, OzBioscience are some of them.

Polyplus-transfection has headquarters located in Illkirch, near Strasbourg and the company’s R&D team has expertise in organic chemistry, polymer and lipid synthesis, cell and molecular biology, and virology. The company says it is currently planning to expand its team and its facilities to better support its customers.

GMP-grade viral vector production

According to Karsten Wilking, Polyplus-transfection’s chairman & CEO, the introduction of the company’s latest transfection reagent, PEIpro-GMP, confirms its place as a leader in transfection reagent technology for cell and gene therapy: “Polyplus-transfection is able to provide a product range that covers the needs of its customers from process development all the way to commercialisation, answering the current key challenges in viral vector production including meeting regulatory requirements, improving manufacturing processes and production scale-up,” she says.

Polyplus-transfection’s PEIpro products provide delivery of plasmid DNA and co-delivery of several-plasmid DNA for large-scale virus production for clinical trials and commercial applications. The virus types produced are mainly adenovirus, adeno-associated viruses (AAV), lentiviral vector (LV), influenza virus, retrovirus, and virus-like particles (VLP) and cell types include the mammalian producer cell lines HEK 293 and its derivatives; BHK, VERO cell lines and virus-specific packaging cell lines, all grown in adherent or suspension cultures.

PEIpro-GMP is the highest-quality grade of PEIpro for therapeutic virus production: it is supplied as a GMP-compliant raw material for human advanced-therapy medicinal products in bags with MPC connectors and weldable tubing for ease of use in closed systems, giving guaranteed virus production yields equivalent to those of the company’s PEIpro or PEIpro-HQ products, thereby easing the risk management process.

A litre of PEIpro-GMP is sufficient to transfect on average 500 litres of cell culture. All product is supplied with a Certificate of Analysis, a Certificate of Compliance, a TSE/BSE Statement and a Non-Hazardous Product Statement, and the following documentation is also available: a DMF (Drug Master File) on file with the FDA; a CMC (chemistry, manufacturing and control) section; a protocol for incoming testing; and a quality agreement. Reagents can be stored at 5+3°C for two years from the date of manufacture.

Validated manufacturing process

PEIpro-GMP is manufactured according to a validated manufacturing process that complies with GMP guidelines to ensure traceability from starting material to the final product. GMP guidelines for manufacturing of ATMP requires that raw materials be of pharmaceutical grade when available (ICH Q7 and Eudralex Vol 4, Part II, Annex I) and, to address this requirement, both steps in PEIpro-GMP manufacturing, ie chemical product and fill & finish, are managed in compliance with GMP guidelines in GMP-accredited facilities. The full list of quality controls assessing identity, potency, purity and safety performed according to the European Pharmacopeia is available upon request from Polyplus-transfection.

Closed systems and single-use technologies are becoming a prerequisite for clinical trials and commercial manufacturing of viral vectors and Polyplus-transfection provides PEIpro-GMP in bags allowing sterile connection compatible with closed systems. Each PEIpro-GMP bag is fitted with one tubing made of thermoplastic elastomer (TPE) that can either be welded or used with MPC connectors, ensuring compatibility with all stirred-tank and fixed-bed bioreactors. With PEIpro-GMP bags, the preparation of large-scale complexes is simplified and guaranteed sterile with a simple ‘plug-and-transfect’ closed system, which considerably reduces the risk of contamination.

Gene therapy is “booming”

According to Wilking, gene therapy has been booming in the past few years, with the use of more efficient and safer viral vectors, and notes that the current bottleneck for the extended use of gene therapy is the cost of the treatment, meaning that this therapy is currently mostly used for rare life-threatening disorders, such as spinal muscular atrophy.

As a result of this “boom” and the business potential it represents, Polyplus-transfection is investing heavily in its R&D activities to support customer needs by improving the manufacturing process as well as reducing manufacturing costs by developing improved and more efficient transfection products. The company continues to work with CDMOs, pharmaceutical companies and biotechs involved in gene therapy and the cell therapy arena worldwide and also partners with other suppliers in the manufacturing chain.

Wilking says that all the external reports Polyplus-transfection has access to confirm “the enormous speed the life sciences market is accelerating at”, with gene therapy specifically delivering significant hope to cure diseases that were up until now seen as untreatable: “We are therefore extremely excited that some of our customers represent the spearhead of this development and that we can play a major role in helping them to improve their patients’ lives,” she concludes.

For further information on Polyplus-transfection visit www.polyplus-transfection.com

Last modified onSaturday, 20 April 2019 07:34