Revolutionizing cell therapies with base editing and AI-engineered CRISPR-Cas enzymes.

In recent years, the field of cellular immunotherapy has witnessed remarkable advancements, including the development of allogeneic CAR-T cells. These engineered cells hold the promise of transforming cancer treatment by harnessing the body's immune system to target and destroy cancer cells. To improve the efficacy of these cellular immunotherapies, complex cell engineering techniques are being adopted.

Advancing allogeneic cell therapies with base editing

Allogeneic CAR-T cells are engineered from donor cells and can be used to treat multiple individuals, making them a more scalable and cost-effective option compared to autologous CAR-T cells, which are derived from the individual's own cells. The Pin-point™ base editing platform enables the generation of multigene edited allogeneic CAR-T cells in a single intervention, helping to overcome the deleterious effects of double-strand DNA breaks (DSBs) and reduce manufacturing timelines.

Harnessing base editing and AI for complex gene edits

Which editing technique to use is a key consideration facing scientists as they seek to avoid the unintended consequences that can occur from gene editing. Traditional CRISPR/Cas9 methods rely on DSBs, which can lead to genomic rearrangements and cytotoxicity, especially when multiple and complex edits are required. The Pin-point platform is designed to make single nucleotide changes in the genome without causing DSBs, which facilitates complex cell therapy engineering in a safer manner.

The Pin-point platform is a modular technology which allows for multiple configurations, including incorporation of the new open-source, AI-generated gene editor enzyme called OpenCRISPR-1 from Profluent. The capability to incorporate the OpenCRISPR-1 enzyme provides additional flexibility and choice to scientists using the Pin-point platform.

Optimizing Gene Editing with AI

The compatibility of OpenCRISPR-1 and the Pin-point platform is an exciting advancement. Bioinformatics is used to design optimal guide RNA (gRNA) sequences for base editing knockout, providing high precision and efficiency.

Conclusion

By providing precise and efficient gene editing with minimal risk of genomic alterations, the combination of the Pin-point base editing platform with AI-engineered CRISPR-Cas enzymes has the potential to help transform cancer treatment. As research and development in this area continue to advance, we can expect to see more innovative applications and breakthroughs.

Find out more in our scientific poster titled: One-step engineering of allogeneic cell therapies with Pin-point base editing platform and an AI-engineered Cas enzyme.