Human embryonic kidney cell lines and their use in the production of rAAV
The application of human embryonic kidney (HEK) cell line and its offspring has solved the problem of low rAAV production efficiency, achieving high-efficiency rAAV packaging and production, and meeting the needs of high-dose gene therapy.
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- SKYLINE THERAPEUTICS (SHANGHAI) CO LTD
- Filing Date
- 2024-09-05
- Publication Date
- 2026-06-02
AI Technical Summary
In the existing technology, the production efficiency of recombinant adeno-associated virus (rAAV) is low, making it difficult to meet the needs of high-dose gene therapy, especially when packaged in mammalian cells, where there is insufficient efficiency.
A human embryonic kidney (HEK) cell line and its progeny are provided, which are capable of efficiently packaging rAAV by systematically transfecting cells with a plasmid containing the rAAV genome, CAP and/or REP peptides encoding AAV, and helper plasmids, and culturing and lysing the cells under appropriate conditions to harvest rAAV.
It has achieved efficient production of rAAV, and even after 90 generations of passaging, it still maintains a high yield, meeting the needs of high-dose gene therapy.
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