Editas Medicine. has been granted a patent for a CRISPR-based genome editing system that targets the HBG1 and HBG2 loci and the BCL11A gene enhancer. The invention includes a ribonucleoprotein complex designed to enhance fetal hemoglobin expression in cells. GlobalData’s report on Editas Medicine 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 Editas Medicine, CRISPR genome editing was a key innovation area identified from patents. Editas Medicine's grant share as of June 2024 was 18%. Grant share is based on the ratio of number of grants to total number of patents.
Crispr methods for increasing fetal hemoglobin expression
The granted patent US12031132B2 describes a ribonucleoprotein (RNP) complex designed for genome editing, specifically targeting the gamma-globin (HBG) gene. The RNP complex consists of a modified Cpf1 variant, which may include mutations, nuclear localization signals, and purification tags, alongside a guide RNA (gRNA) that features specific DNA extensions and chemical modifications. The gRNA is engineered to bind to complementary sequences within the promoter region of the HBG gene, located on chromosome 11. The patent outlines various sequences for both the Cpf1 variant and the gRNA, emphasizing their roles in facilitating targeted genetic alterations to enhance fetal hemoglobin (HbF) expression.
Additionally, the patent details methods for utilizing the RNP complex in ex vivo applications, particularly for modifying hematopoietic stem cells or CD34+ cells. The methods involve delivering the RNP complex to these cells, potentially through electroporation, to induce changes in the HBG gene promoter. This approach aims to increase HbF levels, which could alleviate symptoms associated with sickle cell disease. The patent also specifies the sequences involved in the targeting domain of the gRNA and the Cpf1 variant, providing a comprehensive framework for researchers and clinicians interested in gene editing applications related to 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
