Ultragenyx Pharmaceutical. has been granted a patent for engineered recombinant adeno-associated virus (rAAV) packaging and producer cell lines. These cell lines exhibit reduced expression of the KCNN2 gene, enhancing rAAV titers, with methods for their generation also detailed in the patent. 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 June 2024 was 28%. Grant share is based on the ratio of number of grants to total number of patents.
Engineered cell lines for enhanced raav production
The patent US12018288B2 describes a recombinant adeno-associated virus (rAAV) packaging and/or producer cell line characterized by engineered cells that exhibit reduced expression of the Potassium Calcium-Activated Channel Subfamily N Member 2 (KCNN2) gene compared to their unmodified parental counterparts. The claims further detail that these engineered cells may also show diminished expression of several other gene products, including those from Repulsive Guidance Molecule BMP Co-Receptor A (RGMA) and various other genes. The modifications can involve gene disruptions, partial deletions, or complete deletions of specific genes, enhancing the potential utility of these cell lines in rAAV production.
Additionally, the patent outlines methods for generating these producer cell lines by delivering rAAV vectors to the engineered cells, which can include HeLa or HEK 293 cell lines. The methods aim to improve the production of rAAV when these cells are subsequently infected with a helper virus. The claims emphasize the use of advanced genome editing techniques, such as CRISPR, to achieve the desired gene expression modifications, thereby potentially increasing the efficiency of rAAV production compared to traditional parental cell lines. The patent thus presents a significant advancement in the field of gene therapy and viral vector production.
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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
