Columbia Technology Ventures

Highly efficient T-cell base editing for enhanced immunotherapies

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.

Unmet Need: Improving T-cell therapies and reducing toxicity

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.

The Technology: Advanced base editing for precise and efficient T-cell therapies

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.

Applications:

  • Improved CAR T-cell therapies through precise base editing
  • Development of precision T-cell therapies for autoimmune diseases and cancer
  • High-throughput screening of gene targets
  • Research tool for studying gene editing in T cells

Advantages:

  • Reduced toxicity in T-cell-based therapies
  • Enhanced precision and efficiency in base editing
  • Suitable for large-scale and high-throughput applications
  • Easily integrated for combination therapies

Lead Inventor:

Benjamin Izar, M.D., Ph.D.

Patent Information:

Patent Pending

Related Publications:

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