Beam Therapeutics. has been granted a patent for methods and compositions that edit mutations linked to hemoglobinopathies, including sickle cell disease. The invention utilizes modified adenosine base editors, achieving over 60-70% efficiency, to correct specific mutations in the HBG1/2 promoter, enhancing gamma globin expression. GlobalData’s report on Beam Therapeutics 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 Beam Therapeutics, CRISPR genome editing was a key innovation area identified from patents. Beam Therapeutics's grant share as of June 2024 was 4%. Grant share is based on the ratio of number of grants to total number of patents.
Editing mutations for treating sickle cell disease
The granted patent US12016908B2 outlines a novel method for treating sickle cell disease or beta-thalassemia through the administration of genetically edited cells. The method involves the use of a cell containing an edited hemoglobin subunit gamma 1 and/or 2 (HBG1/2) promoter, specifically modified to include an A to G alteration at positions 5 and/or 8 of a designated nucleotide sequence. This editing is achieved by employing a guide RNA and a base editor that consists of a programmable DNA binding domain and an adenosine deaminase domain. The adenosine deaminase domain must exhibit at least 95% sequence identity to a specified amino acid sequence and can include specific amino acid alterations, such as arginine or threonine at position 147. The resultant deamination process enhances gamma globin (HbF) expression in the treated subject compared to untreated individuals.
Further claims detail the types of cells that can be utilized, including hematopoietic stem cells and various progenitor cells. The patent also specifies the potential configurations of the adenosine deaminase domain, including combinations of amino acid alterations. The guide RNA used in the process is defined by a specific nucleotide sequence, and the method may involve the use of a dead Cas9 (dCas9) or a nickase Cas9 (nCas9) as part of the base editor. Additionally, the patent describes the possibility of using autologous cells, particularly CD34+ hematopoietic stem cells, to ensure compatibility with the subject. Overall, the claims present a comprehensive approach to gene editing aimed at increasing HbF levels, which could significantly impact the treatment of these hemoglobinopathies.
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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
