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European Biopharmaceutical Review

CRISPR: The Jump From Bench to Clinic

CRISPR and other genome editing tools have shown great promise as new therapeutics in vitro. The technology has advanced rapidly, and the potential for life-changing therapies has been demonstrated over and over. The real problem facing CRISPR, and all genome editing tools now, is one of scalability and reproducibility. What has been shown in the laboratory now needs to be robustly demonstrated on much larger scales and the results must be the same every single time.

Not only that, the leap from in vitro lab bench to in vivo clinical studies is huge. Performing a CRISPR experiment in a cell culture dish lacks most of the complexity of human cells embedded in a complex tissue system. We have to be prepared to encounter and overcome these problems if genome editing is to become a real therapeutic option in the future. We have been down this road to a certain extent with small molecule drugs for cancer and biologics. We know that factors such as tumour microenvironments, immune responses, and tissue hypoxia can drastically alter how cells respond to treatment. CRISPR therapies are relatively untested in these complex scenarios, which needs to change for them to be considered as broad-scale and viable therapy options.

If the complexity was not problem enough, the final hurdle is scale. A drug is not developed if it cannot be manufactured and mass produced, meaning we need to start thinking this way about CRISPR. How do we really scale up the therapies so that they can be widely used to treat disease?

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Cosimo Ducani PhD is the CEO and Co-Founder of Moligo Technologies. He was previously a post-doctoral fellow at the Karolinska Institute, Sweden, where he researched DNA nanotechnology. Dr Ducani was a Marie Curie Early Stage Researcher at the University of Birmingham, UK, studying the interactions of synthetic drugs and proteins. His work has often centered on the intersection of synthetic biology and DNA nanotechnology. Dr Ducani received fellowships from the Hagelén Foundation and the Swedish Society for Medical Research. He has a Doctorate in Applied Chemistry from the University of Bari, Italy, and Bachelor’s and Master’s degrees in Biotechnology from the University of Salento, Italy
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Cosimo Duncani
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