Gene therapy approach consists in introducing a sequence of DNA into the cells or tissue of an individual to repair a genetic default – which can be inherited from parents or induced by the environment – which leads to severe diseases and death.
Gene therapy field encompasses many different products and their uses. It includes many kinds of virus-based vectors such as AAV serotypes and lentivirus vectors. AAV derived products are classically used to directly cure genetic diseases such as Duchenne myopathy, cystic fibrosis, or haemophilia… A number of vaccinia-based vectors may be used against cancer to target and specifically kill these tumor cells. Lentiviruses are currently used to generate CAR-T cells by ex vivo modifying cells from patients suffering of cancer – such as leukaemia – before reinjecting them as a treatment to enhance an immune anti-tumor response.
Clean Cells has a strong track record of working with the gene therapy industry, thanks to its extensive regulatory knowledge and GMP license. It offers a diverse range of dedicated services, including cell bank manufacturing, characterization and testing, and storage services. Clean Cells’ expertise in gene therapy services enables assisting their clients in advancing their gene therapy products from preclinical development to clinical trials and ultimately to commercialization.
Clean Cells has extensive expertise in gene therapy field which applies to both research and development (R&D) and GMP-compliant biomanufacturing settings. The company provides advanced services, which include mammalian (Human cells HEK293) and insect (Sf9, Sf21) cell banking to support R&D and GMP processes.
Clean Cells is an established authority in gene therapy dedicated services with extensive experience in biologics biosafety testing. As a market leader, the company has a proven track record in cGMP expertise and its team has developed and validated a variety of assays applicable to the entire range of gene therapy products.
Moreover, Clean Cells has developed a range of GMP compliant assays for lentivirus products and for AAV products, including respectively recombinant lentivirus (RCL) and adeno-associated virus (rcAAV) assays.
| Available Tests | BH/UnPB | DS | DP |
| Replication-competent AAV particles (rcAAV) or Lentiviral particles (RCL) detection | • | (•) | |
| Vector genomes titration | • | • | (•) |
| Infectious particles titration | • | • | • |
| Physical particles titration | • | • | • |
| Residual genomic DNA | (•) | • | |
| Residual plasmid DNA | (•) | • | |
| Residual Host Cell Proteins | (•) | • | |
| Identity (sequencing) | • | • | |
| Vector genome transfer efficiency, gene of interest expression levels (mRNA and protein) |
• | • | |
| Biological activity | • | • |
Through its testing options, Clean Cells ensures quick turnarounds and fewer volumes for testing. The company stays abreast of regulatory frameworks to establish customized gene therapy testing services, as required by the EMEA and FDA.
To develop safe and effective gene therapies, tailored services are crucial for certain gene therapy products. Clean Cells offers a comprehensive range of gene therapy services, including analytical development solutions for various biomolecular tests and bioassays, culture tests, immunoassays, and NGS.
Clean Cells also assists with the qualification and validation of analytical methods for gene therapy products. This may involve conducting initial qualification, transferring methods in-house, or integral validation of assays in compliance with ICHQ21 and cGMP guidelines as part of a gene therapy program.
In gene therapy production, cell banks are critical components ensuring safe and consistent material in upstream and downstream processing. Our integrated gene therapy services provide cell bank storage solutions to ensure the security and stability of R&D and GMP materials.
Our storage areas are equipped with 24/7 surveillance to offer the highest security for your valuable gene therapy materials.
For additional information on gene therapy services and how they apply to your product, please get in touch with our team of experts.
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