Vertex Pharmaceuticals. has been granted a patent for methods and materials aimed at treating hemoglobinopathies, including sickle cell anemia and ß-thalassemia. The patent covers genetically modified progenitor cells that enhance fetal hemoglobin production through genome editing techniques involving Cas9 endonuclease and guide RNAs. GlobalData’s report on Vertex Pharmaceuticals 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 Vertex Pharmaceuticals, Human telomerase RT biomarker was a key innovation area identified from patents. Vertex Pharmaceuticals's grant share as of June 2024 was 44%. Grant share is based on the ratio of number of grants to total number of patents.
Genetically modified cells for treating hemoglobinopathies
The patent US12043843B2 describes a genetically modified human cell that incorporates specific genetic alterations aimed at enhancing the expression of ?-globin and fetal hemoglobin (HbF). The modification involves the deletion or insertion of chromosomal sequences complementary to a series of specified nucleic acid sequences (SEQ ID NOs). The process utilizes a Cas9 endonuclease and one or more guide RNAs (gRNAs) to create double-strand breaks at targeted loci within the dß-globin region of human chromosome 11. This genetic engineering approach is particularly relevant for cells derived from patients with ß-hemoglobinopathies, including various forms of sickle cell disease and thalassemia.
The claims further detail the characteristics of the modified cells, including the potential for increased expression of ?-globin and HbF compared to unmodified cells. The modified cells can be isolated progenitor cells, such as hematopoietic progenitor cells or induced pluripotent stem cells. Additionally, the patent specifies that the modifications can occur at loci near known deletions associated with hemoglobin disorders. The invention also encompasses a composition that includes a plurality of these modified cells combined with a pharmaceutically acceptable carrier or salt, indicating potential therapeutic applications in treating hemoglobin-related diseases.
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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
