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Immitra Bio has developed a proprietary gene-integration platform, HematoGen, which combines CRISPR-Cas9 gene editing with hematopoietic stem cell technology to enable precise and durable correction of genetic defects in blood stem cells. The company’s approach aims for lifelong therapeutic benefit by addressing the root cause of rare genetic diseases. Their programs are currently at the pre-clinical stage and focus on haemoglobinopathies such as beta-thalassemia and alpha-thalassemia[1][4].
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