Methods for the clinical-scale production of genetically modified primary cells

The use of a high-volume gas-permeable cell culture device and flow-through electroporation system addresses DDR and efficiency issues in genome editing, enhancing cell viability and reducing risks in primary cell therapies.

JP2026518451APending Publication Date: 2026-06-08KAMAU THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
KAMAU THERAPEUTICS INC
Filing Date
2024-05-31
Publication Date
2026-06-08

AI Technical Summary

Technical Problem

Current methods for genome editing in primary cells, particularly via the homologous recombination (HR) repair pathway, face challenges such as DNA damage response (DDR) and reduced efficiency, necessitating high doses of genetically modified cells, which are costly and risky, especially for therapies like autologous hematopoietic stem and progenitor cell (HSPC) therapy.

Method used

A process utilizing a high-volume gas-permeable cell culture device and flow-through electroporation system to improve gene editing performance, enhance cell yield, and reduce DDR, involving culturing primary cells with a gas-permeable membrane and electroporating them in a closed chamber to facilitate efficient gene editing and cell viability.

Benefits of technology

The process achieves improved double-strand break formation, increased HR and NHEJ frequencies, enhanced cell viability, and reduced manufacturing time, while minimizing the need for viral DNA donors, thus improving the safety and efficacy of gene-edited cell therapies.

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Abstract

The process provided in this invention transfects primary cells with gene editing reagents using a high-volume gas-permeable cell culture device and a flow-through electroporation device under conditions that improve gene editing performance, cell yield, and drug (DP) quality characteristics for cell therapy applications. As demonstrated in the examples, primary cells edited according to the process provided herein achieved improved double-strand break (DSB) formation rates, increased frequency of homology-directed repair (HR) and non-homologous end joining (NHEJ) combinations, increased frequency of bi-allelic and mono-allelic HR events, improved cell viability, proliferative capacity, and cell fitness after gene editing, and reduced manufacturing time.
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