Scientists Develop AI-Driven Gene Editing Tool with Enhanced Precision for Human Diseases and Gene Therapies

Researchers at the University of Zurich have developed a new method that uses artificial intelligence to improve the precision of genome editing with CRISPR/Cas technology. The method, called Pythia, predicts how cells repair DNA after it is cut and guides the cell to make precise genetic changes using small DNA repair templates. This approach was tested in human cell cultures, Xenopus, and living mice, showing highly accurate gene edits and integrations. The technique has the potential to improve modeling of human diseases and lay the groundwork for next-generation gene therapies.

Researchers at the University of Zurich have developed a novel method called Pythia, which leverages artificial intelligence to enhance the precision of genome editing with CRISPR/Cas technology. This innovative approach uses AI to predict how cells repair DNA after it is cut, guiding the cell to make precise genetic changes via small DNA repair templates. The technique was successfully tested in human cell cultures, Xenopus, and living mice, demonstrating highly accurate gene edits and integrations.

Pythia, named after the high priestess of the oracle at the Temple of Apollo of Delphi, allows scientists to forecast the outcomes of gene editing with remarkable precision. By simulating millions of possible editing outcomes using machine learning, the AI-designed templates enable highly accurate genetic modifications and integrations. This method can also be used to fluorescently label specific proteins, providing direct observation of protein activity in both healthy and diseased tissue.

The technology's ability to work well in all cells, including those in organs with no cell division like the brain, opens up new possibilities for understanding genetic diseases and developing safer, more effective gene therapies. This breakthrough not only improves the accuracy of genetic modeling but also lays the groundwork for next-generation gene therapies.

The development of Pythia comes at a critical time as CRISPR-based gene therapies are gaining traction in the market. CRISPR Therapeutics, for instance, has seen significant sales growth with its one-shot gene therapy, Casgevy, which was approved for two blood disorder indications in 2023 and 2024. Casgevy recorded sales worth $30.4 million in the second quarter of 2025, reflecting a 114.1% sequential increase and underscoring strong market adoption. The global launch of Casgevy continues to gain momentum, with more than 75 authorized treatment centers activated across all regions where the therapy is approved.

The success of Casgevy highlights the potential of CRISPR-based therapies, which are expected to secure a reliable revenue base for CRISPR Therapeutics and enable greater investment in its gene-editing pipeline. Continued demand for Casgevy may also broaden revenue sources and strengthen the company’s long-term growth prospects in the competitive cell and gene therapy market.

In conclusion, the development of Pythia by the University of Zurich represents a significant advancement in genome editing technology. By enhancing the precision and predictability of CRISPR/Cas-based gene editing, this AI-driven method opens new avenues for the development of safer and more effective gene therapies, with potential applications in various genetic diseases.

References:
[1] https://phys.org/news/2025-08-ai-crispr-precise-gene.html
[2] https://www.nasdaq.com/articles/can-rising-casgevy-sales-aid-crispr-therapeutics-post-q2-earnings