SBiomedics (CEO Dong-Wook Kim and Se-Il Kang) recently announced a new treatment technology with improved safety and efficacy related to the cell and gene therapy development technology for hemophilia A, an intractable genetic disease currently in progress, through the international academic journal EMM (Experimental & Molecular Medicine). EMM is an internationally renowned biomedical science journal published by Nature Publishing Group (NPG), and the research was conducted jointly with Yonsei University.
Through this presentation, SBiomedics introduced the results of its research and development of cell and gene therapies for intractable genetic diseases, which will become a new future pipeline, in addition to the development of a Parkinson's disease treatment currently undergoing clinical trials.
Hemophilia A is a rare genetic disease that causes bleeding that does not stop easily due to a deficiency of clotting factor VIII due to a mutation in the blood clotting factor VIII gene (FVIII). It affects more than 400,000 people worldwide. Currently, there is no fundamental cell-based treatment, and methods that mainly supplement recombinant blood clotting factor proteins are used, and AAV vector-based gene therapy has been developed, but it is expensive.
In this research paper, we successfully introduced a strategy to insert and correct the 8th gene with improved function using safer gene scissors into human induced pluripotent stem cells (iPS cells) derived from hemophilia patients.
The research team developed a strategy to correct genes more safely than previous technologies without creating indels (insertion + deletion) by using a mutant gene scissors (Cas9 D10A nickase) that cuts only one strand of the chromosome rather than the existing gene scissors. In addition, in the case of the therapeutic gene, the team presented a new strategy that simultaneously improved clotting activity and safety by using a mutant clotting factor 8 with improved function and inserting it into a safe part of the chromosome.
For preclinical studies, the research team differentiated gene-corrected stem cells into vascular endothelial cells that produce blood clotting factors, and transplanted them into hemophiliac mice. As a result, mice that received gene-corrected cells produced blood clotting factors, and bleeding symptoms were significantly improved.
Kim Dong-wook, CEO and Chief Technology Officer of SBiomedics (and concurrently a professor at Yonsei University College of Medicine), said, “This paper presentation is part of the company’s R&D on cell and gene therapies for intractable diseases that we are focusing on in preparation for the future.” He also expressed his aspirations, saying, “Rather than being a company that stops at commercializing treatments for intractable diseases, including cell therapies for Parkinson’s disease that are currently in clinical trials, we will become a company that continuously develops better treatments through continuous research on future technologies.”
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