Ultragenyx Pharmaceutical has been granted a patent for a method involving HDAC inhibitors and AAV rep proteins to culture cells, generate cell lines, and deliver polynucleotides. The method includes integrating a recombinant AAV vector into chromosome 19 of HeLa cells to produce rAAV at a high titer. GlobalData’s report on Ultragenyx Pharmaceutical gives a 360-degree view of the company including its patenting strategy. Buy the report here.
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According to GlobalData’s company profile on Ultragenyx Pharmaceutical, Adeno-associated virus vectors was a key innovation area identified from patents. Ultragenyx Pharmaceutical's grant share as of February 2024 was 27%. Grant share is based on the ratio of number of grants to total number of patents.
Method of developing stable cell line for producing raav
A recently granted patent (Publication Number: US11898170B2) discloses a method for developing a stable cell line capable of producing recombinant Adeno-Associated Virus (rAAV) at a high titer. The method involves integrating a specific rAAV vector sequence into chromosome 19 of HeLa cells, culturing the cells under suitable conditions, and identifying cells with the integrated vector. The resulting cell line is capable of producing rAAV at a titer of at least 8.5×1010 GC/ml after four days of culture. Various techniques such as electroporation, chemical-based transfection, microinjection, or cell squeezing can be used for delivering the rAAV vector and rep mRNA into the cells.
Furthermore, the patent claims cover the integration of the rAAV vector sequence into a specific region on chromosome 19, the optimal amount of rep mRNA to be provided to the cells, and the range of rAAV titers achievable with the developed cell line. The method specifies the use of HeLa S3 cell culture for the process and also includes the generation of a stable HeLa cell line and a multitude of rAAV particles as part of the claimed invention. The patent provides a detailed and specific method for efficiently developing a cell line capable of producing high titers of rAAV, which can have significant implications in the field of gene therapy and biotechnology.
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GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies
