Making CRISPR more accessible for scientific innovation
At ERS Genomics, we make CRISPR/Cas9 accessible to innovators. By licensing the foundational IP co-owned by Professor Emmanuelle Charpentier, we enable a broad spectrum of applications outside of direct therapeutic use—from drug discovery to innovation in fields far beyond pharma. One of our licensees, Bioneer A/S, exemplifies how CRISPR is shaping modern drug discovery as an essential tool.
Bioneer: A trusted partner in drug discovery
Bioneer is a strategic partner to a broad range of biotech and pharmaceutical companies globally. They have a 40-year proven track record of delivering impact to the companies by supporting their drug discovery phases. Bioneer offer services within assay development and disease modelling as part of their drug discovery expertise. Headquartered in Hørsholm, Denmark, Bioneer operates a 3,350-square-meter facility equipped with advanced laboratories.
Harnessing CRISPR for advanced disease modelling
Among their many research tools, Bioneer uses CRISPR/Cas9 to develop precise, cell-based models for drug discovery. The ability to edit genes with precision is key to studying diseases and testing new drug candidates. CRISPR provides a direct way to introduce or correct genetic mutations in human cells, making it possible to model diseases and recapitulate disease phenotypes.
One of Bioneer’s key applications of CRISPR is the creation of genetically modified cell lines for in vitro disease modelling. These models help Bioneer & their customers to study genetic disorders and test drug candidates under controlled conditions. CRISPR makes it possible to insert or remove single base pairs, generating isogenic cell pairs that allow researchers to pinpoint the effects of specific mutations. This level of precision is invaluable for uncovering how genetic changes drive disease.
Beyond single-gene modifications, Bioneer also applies CRISPR for gene knock-outs, selectively removing parts of a gene to stop protein production. This is a crucial technique for studying gene function and identifying potential drug targets. More complex modifications, like deleting or inserting specific sequences, help refine models to better reflect disease-related genetic alterations.
Another important application is the development of reporter cell lines. By inserting fluorescent markers such as GFP, scientists can visualize gene expression and cellular activity in real-time, improving drug screening and functional studies. Bioneer also uses CRISPR to create inducible gene expression systems, allowing researchers to switch genes on or off in a controlled way—an essential tool for studying dynamic biological processes.
Bioneer´s use of CRISPR in neurological disease research
CRISPR is a central tool for the CNS Assay development team at Bioneer. Human neurons are an important tool for studying neurological diseases like Alzheimer’s and Parkinson’s. By using CRISPR to introduce specific genetic mutations, Bioneer develop more accurate disease models, helping companies to explore how these conditions develop at the cellular level.
To enhance model complexity, Bioneer has developed co-culture systems that combine neurons with glial cells, such as microglia and astrocytes. These models better reflect the cellular interactions of the human brain, making them more relevant for studying neurodegeneration and neuroinflammation. CRISPR allows for precise genetic modifications in these systems, making it possible to isolate and analyze the roles of individual genes in disease progression.
Enabling responsible innovation with a CRISPR License from ERS Genomics
The CRISPR license Bioneer holds from ERS Genomics grants them access to the foundational CVC patents, which cover the core aspects of CRISPR/Cas9 gene editing. With over 130 patents globally, including more than 50 in the U.S ., these rights are essential for conducting CRISPR-based research legally and effectively. By securing a license, Bioneer ensures they can continue offering CRISPR-based services while adhering to the intellectual property framework that supports responsible innovation. This also provides confidence to their customers and partners that the IP foundations of their work are secure and futureproofed.