Early preclinical studies highly rely on predictive cell models. Bioneer develops a range of 3D cell models based on human cells combined with gene editing technology.
Our current R&D program within Disease Models focuses on using novel gene editing technologies and advanced cell culture systems to develop a panel of disease models.
The R&D group is located at the Bioneer headquarter facilities, where the gene editing and iPSC core facility is a central infrastructure for the R&D program.
Modelling of diseases is an important tool for the finding of new lead drugs and understanding of their mode of action. Bioneer has for several years been working in the field of disease models from both immortalized cell lines and especially via induced Pluripotent Stem Cells (iPSC). Bioneer has established all facilities for the generation of iPSCs, for their quality control analysis and the following differentiation into specific cell type for modelling.
In order to satisfy requirements for documentation to regulatory bodies of critical aspects of drug candidates there is an increasing demand for early preclinical studies based on cell models where e.g. MoA and toxicological effects can be analysed. Bioneer develops a range of 3D cell models based on human iPS cells. We have currently focus on tox assessment and the cytochrome P450 drug metabolizing enzymes . In addition, we have started to develop 3D skin models for validation of drug candidate molecules including cosmetic products.
In order to be able to validate our generated 3D disease models, we are currently implementing our ICH competences on 3D models of liver cells and neurons. Bioneer is implementing visualization technology of relevant biomarkers for the documentation of our 3D disease models.
Bioneer has large experience in building neurodegenerative models based on patient specific induced Pluripotent Stem Cells (iPSC). To advance these models to express more mature in vivo like phenotypes, differentiation in 3D organoid structures are exploited.
Several Alzheimers disease (PSEN1, APP, APOE) and frontotemporal dementia (MAPT, CHMP2B, GRN) models are being perused. In addition, to enhance the differentiation to specific neuronal subtypes, Bioneer is utilizing systems based on controlled expression of specific transcriptions factors.
Gene editing technology has turned out to be the new breakthrough in tailored DNA engineering for research and target validation. Using custom designed and sequence specific double DNA strand cutting enzymes, like the CRISPR/CAS9 system or TALENs, gene engineering in human cells has become an elegant tool to generate new disease models. Bioneer has during the last few years build a whole collection of isogenic cell lines to study the phenotype of disease causing mutations. Many of these lines are stored in the European Bank for induced pluripotent Stem Cell, EBiSC.
> Cryopreservation and Large-Scale Screening
Bioneer has initiated a strategic collaboration with Frauenhofer Institute for Biomedical Engineering to implement solutions covering needs for automated processing of living cells by jointly developing technology for storage and large-scale automated cell based screening in 3D.
> European Bank for Induced pluripotent Stem Cells
Bioneer has since 2014 been part of the European Bank for induced pluripotent Stem Cells (EBiSC). EBiSC is a large European public-private partnership project coordinated by Pfizer Ltd and managed by Roslin Cells Siences Ltd. and supported jointly by the Innovative Medicines Initiative (IMI) and members of the European Federation of Pharmaceutical Industries and Associations (EFPIA). The purpose of EBiSC is to establish a robust, reliable supply chain from the generation of human iPS cell lines, covering many different diseases. The bank builds on internationally accepted quality criteria and their resources are available worldwide to any qualified user. Read more here.
Bioneer is part of BrainStem, which has been granted 24 MDKK by the Innovation Fund Denmark and will do research in neurodegenerative diseases over a 6 year period – from 2015 until 2020. BrainStem is a Stem Cell Center of Excellence in Neurology. To learn more about the project, read here.
Rasmussen, MA., Holst, B., Tümer, Z., Johnsen, MG., Zhou, S., Stummann, TC., Hyttel, P., Clausen, C. (2014) Transient p53 Suppression Increases Reprogramming of Human Fibroblasts without Affecting Apoptosis and DNA Damage. Stem Cell Reports 3, 404-413
EBiSC, Innovative Medicines Initiative project:
De Sousa PA. et al. (2017) Rapid establishment of the European Bank for induced Pluripotent Stem Cells (EBiSC) – the Hot Start experience. Stem Cell Research 20, 105-114
- Zhang, Y., Schmid, B., Nikolaisen, NK., Rasmussen, MA., Aldana, BI., Agger, M., Calloe, K., Stummann, TC., Larsen, HM., Nielsen, TT., Huang, J., Xu, F., Liu, X., Bolund, L., Meyer, M. Bak, LK., Waagepetersen, HS., Luo, Y., Nielsen, JE., The FReJA Consortium, Holst, B., Clausen, C., Hyttel, P., Freude, KK. (2017) Patient iPSC-Derived Neurons for Disease Modeling of Frontotemporal Dementia with Mutation in CHMP2B. Stem Cell Reports 8, 1-11
- Ochalek et al. (2017) Neurons derived from sporadic Alzheimer’s disease iPSCs reveal elevated TAU hyperphosphorylation, increased amyloid levels, and GSK3B activation. Alzheimer’s Research & Therapy 9:90
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