This technology is an ultra-efficient base-editing system for human T cells that can be used to improve therapies for cancer and autoimmune diseases.
Current cellular therapies, such as adoptive T cell transfer and CAR-T cell therapies, are limited by low editing efficiency in primary human T cells, resulting in reduced therapeutic effectiveness. Additionally, these therapies can cause significant toxicity or fail to elicit a sufficient immune response in patients. There is a need for a more efficient, precise, and less toxic genome-editing method to improve the safety and efficacy of cellular therapies for cancer and autoimmune disorders.
This technology provides a highly efficient base-editing system that uses adenine and cytosine base editors in human T cells. Unlike traditional methods that involve viral transduction, this system leverages in vitro transcription to produce a single RNA transcript for delivery. This approach enhances gene editing by using single-guide RNAs to precisely target genes, ensuring timed and controlled edits. This method is scalable, enabling high-throughput, large-scale genomic screens to discover new therapeutic targets for cancer and autoimmune disorders. Additionally, the system supports the generation of both natural and synthetic gene variants to enhance T cell function and reduce treatment-related toxicity.
This technology has been validated with primary human T cells.
Patent Pending (WO/2025/106732)
IR CU24092
Licensing Contact: Joan Martinez