technologies products We develop proprietary technology and licensing partnerships to ensure you have access to best-in-class solutions that add value to your own science. regulatory support In close collaboration with you, Lonza Regulatory Affairs provide a CMC dossier suitable to the stage of clinical development of your product manufactured by Lonza. technical support The collective expertise of our multi-disciplinary team in protein expression and production processes can help you get the most from our industry-leading technologies. knowledge center Gather additional industry and technical information from our repository of resources and educational content.Exosomes are small membrane vesicles that are secreted by a multitude of cell types, which play a role in cell to cell communication. They can serve as clinically valuable tools for early diagnosis, prognosis and potentially targeted treatment.From a manufacturing stand-point, the challenge resides not only in the production but also in the characterization. Exosomes have the potential to make cell and gene therapies commercially viable for large populations.We are still at a very early stage and most developers of these innovative technologies are in pre-clinical or early clinical stages, working to demonstrate the efficacy and the potential of exosomes-based therapies.Prime biological cargo deliveryExosomes are produced by all cells. They are a sub-group of extracellular vesicle with a size range typically between 30 and 150 nm, have a Phospholipid Bilayer shell with specific surface markers, and a genetic package containing of RNA, DNA and proteins.Asset 1Some cells can use exosomes to transfer genetic material from one to another. Specifically, an exosome can transfer microRNAs to control the expression of a gene, and messenger RNAs to manufacture proteins. They also transfer proteins themselves, lipids, DNA and others.Exosomes are acting as cargo delivery systems in our body to deliver specific packages with positive or negative messages from one cell to another in a targeted way at distance. Simply put, they are a fresh and well conserved source of biomarkers coming from live cells which reflect those of the parenteral cells.So what stands in the way?Artboard 3 copy 6Aside from decoding the biology and mechanism for therapeutic effect in this novel field, these are some key challenges faced by exosomes-based therapy developers. Those are linked to the very early and pre-clinical stage which most of the exosomes-based therapies are in. There are limited characterization methods available, a need for precise isolation and purification methods and few partners who can provide access to end-to-end GMP manufacturing expertise. Identifying optimal formulation, obtaining starting cell-lines, media, gene editing cargo loading can be a challenge, as well as defining functional assays.No mountain high enoughChoosing the right partner becomes critical in such a pioneering field. By leveraging our knowledge in 2D/3D cell culture and our viral manufacturing expertise, alongside the latest exosome characterization technologies, we can provide a complete path for exosome manufacturing.CharacterizationA complete set of measurement tools to enable both upstream and downstream process development as well as quality control and product characterization Further characterization techniques currently on trial in R D, targeted mid-2020 for GMP use FACS - FLourescence-activated cell sorting qPCR - Real-time quantitative polymerase-chain-reaction ELISA - enzyme-linked immunosorbent assay Tissue acquisition and cell isolation (or exising cell line) Nucleofection / electroporation: Lonza Nucleofector™ and other electroporation methods Lentiviral and AAV transduction methods Large-scale 2D systems: CellSTACK®s and cell factories Large-scale 3D systems: stir tank bioreactor (adherent and aggregate based cultures), Wave Bioreactor™ (suspension based cultures), hollow fiber (Quantum®) Process parameters to achieve final composition of exosome in bulk DS Shipping and storage End-to-end traceability, identification and injection Selection of appropriate buffer and excipients to have optimal formulatuon for improved stability Development services for lyophilization of exosome for 5°C storage Freeze / thaw and agitation study to support handling and transportation Stability studies, manufacturing compatibility, particle assessment ID testing*, light stability Overfills, container closure system selection qualification, container closure integrity in frozen state* Extractables / leachables testing, in-use clinical administration design (pharmacy manual) and testing GMP DP manufacturing QC release and stability studies (including project specific development) and implementation of Pharmacopeia methods Implementation of purity methods from customer for QC release and stability Leveraging 20+ years experience of cell therapy upstream and viral vector downstream, alongside 100+ iPSC, HEK and MSC customer projects 2+ years of exosome manufacturing expertise, including research and development Exosomes are strategic to our business and we are investing in expanding our capabilities in this area Partnership with Exosomics S.p.A. to enable enhanced characterization technologies Extensive bioprocessing and bioreactor experience for a wide range of applications Development of manufacturing processes using various starting cell types including somatic cells, pluripotent and tissue-specific stem cells End-to-end service offering: Phase appropriate assay and process qualification Full characterization Tissue acquisition and cell line development Upstream, downstream, formulation and fill finish Supply chain orchestration system through partnerships Lifecycle regulatory support Thought leadership through global, dedicated exosomes projects and collaborations Open to a variety of collaboration options with exosomes developers Clinical Manufacturing, Commercial Manufacturing, Cell and Gene Manufacturing, Viral Vector Manufacturing Clinical Manufacturing, Commercial Manufacturing, Cell and Gene Manufacturing, Process Development, Analytical Development All trademarks belong to their respective owners and are only being used for informational purposes.