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 July 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 product compared to unmodified parental cells. The claims further detail that these engineered cells may also show reduced expression of several other gene products, including those from Repulsive Guidance Molecule BMP Co-Receptor A (RGMA) and ATP Synthase F1 Subunit Epsilon Pseudogene 2 (ATP5EP2), among others. The modifications to the gene expression 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 and subsequently infecting them with a helper virus to produce rAAV. The claims specify that the engineered cell lines can be derived from HeLa or human embryonic kidney (HEK) 293 cells, and the reduction of KCNN2 expression can be achieved through various genome editing techniques, including CRISPR. The patent also includes claims for the lysate or cell culture supernatant derived from these producer cell lines, indicating a focus on improving rAAV production efficiency compared to control parental cell lines.
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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
