BioMarin Pharmaceutical has been granted a patent for novel adeno-associated virus (AAV) capsid proteins, AAV particles, and vectors expressing these proteins. The invention includes methods for making these vectors and their therapeutic uses. The patent also covers a recombinant AAV containing specific capsid proteins and a transgene for gene expression in host cells. GlobalData’s report on BioMarin 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 BioMarin Pharmaceutical, Human telomerase RT biomarker was a key innovation area identified from patents. BioMarin Pharmaceutical's grant share as of January 2024 was 40%. Grant share is based on the ratio of number of grants to total number of patents.
Recombinant adeno-associated virus for gene therapy
A recently granted patent (Publication Number: US11821008B2) discloses a novel recombinant adeno-associated virus (rAAV) designed for efficient gene delivery. The rAAV comprises a capsid protein with specific amino acid sequences that are highly identical to certain regions of known sequences, along with a transgene controlled by a regulatory sequence. This composition can be used in methods for delivering genes to cells, particularly liver cells, and treating disorders associated with abnormal protein activity, such as hemophilia A, hemophilia B, Wilson's disease, hereditary angioedema (HAE), alpha 1 antitrypsin deficiency, and galactosemia.
Furthermore, the patent covers methods for producing the rAAV through cell culture techniques using specific cell lines like HEK293, insect cells, or mammalian cells. The resulting rAAV can be collected from the cell culture supernatant and utilized for gene therapy applications. The patent emphasizes the high sequence identity required for the capsid protein, ensuring the efficacy of gene delivery and treatment outcomes. These innovative methods and compositions offer promising avenues for addressing genetic disorders and diseases linked to aberrant protein function, showcasing the potential of rAAV technology in the field of gene therapy.
To know more about GlobalData’s detailed insights on BioMarin Pharmaceutical, buy the report here.
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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
