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Kamau stands apart from the rest of the gene editing field through the unique capabilities of its next-generation gene correction platform. This technology is founded on homology directed repair (HDR) to correct genetic mutations with unprecedented precision through the CRISPR-Cas9 system that precisely cuts pathologic mutations and pastes the correct sequence to restore healthy function. Our HDR platform is built on the groundbreaking research of co-founders Matthew Porteus, M.D., Ph.D. and Maria Grazia Roncarolo, M.D., whose work propelled gene editing to the next level of targeted, high-fidelity gene correction to address the underlying causes of diseases.
We are expanding the field of genome editing by combining the innate power of HDR with CRISPR-Cas9 gene editing. Our next generation platform technology can rewrite genomic DNA to:
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Our novel gene correction approach, called homology directed repair (HDR), overcomes prior limitations in specificity, efficiency, and durability of gene editing to offer broad potential for transforming human health outcomes through the delivery of one-time curative cell therapies. Our lead program, nulabeglogene autogedtemcel (nula-cel) is the only hematopoietic stem-cell based therapy in clinical development for sickle cell disease that precisely corrects the mutation in the beta-globin gene, restoring the patient’s stem cells to normal adult hemoglobin. Our platform aims to extend the application of HDR to treat or cure a range of serious genetic diseases with unmet medical needs.