Universal donor cells

JP2025170191A5Pending Publication Date: 2025-12-17CRISPR THERAPEUTICS AG
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Patent Information

Application Number
JP2025117434
Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2020-02-21
Filing Date
2025-07-11
Publication Date
2025-12-17

AI Technical Summary

Technical Problem

Existing methods for generating universal donor cells face limitations such as susceptibility to natural killer (NK) cell lysis due to lack of HLA expression and residual mRNA, and potential off-target cleavage issues with TALEN technology, which pose safety concerns for clinical use.

Method used

The method involves delivering site-specific nucleases like CRISPR-Cas9 to target genes encoding survival factors (e.g., TXNIP, ZNF143, FOXO1, MANF) and tolerogenic factors (e.g., PD-L1, HLA-E, HLA-G, CTLA-4, CD47) to generate universal donor cells with enhanced immune evasion and survival capabilities, using specific gRNAs to ensure precise gene editing.

Benefits of technology

The generated universal donor cells demonstrate increased immune evasion and survival post-engraftment by reducing MHC-I expression and introducing tolerogenic factors, thereby overcoming NK cell lysis and allogeneic rejection.

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Abstract

To provide genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and to provide methods of generating the genetic modified cells.SOLUTION: The universal donor cells comprise at least one genetic modification within or near at least one gene encoding a survival factor, the genetic modification comprising the insertion of a polypeptide encoding a tolerogenic factor. The universal donor cell may further comprise at least one genetic modification within or near a gene encoding one or more MHC-I or MHC-II human leukocyte antigens or components or transcriptional regulators of the MHC-I or MHC-II complexes, the genetic modification comprising the insertion of a polypeptide encoding a second tolerogenic factor.SELECTED DRAWING: Figure 1
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