Empowered Patient Podcast Using Self-Replicating RNA Technology to Treat Telomere Biology Disorders with Akihiro Ko Elixirgen Therapeutics
Akihiro Ko is the CEO and Co-Founder of Elixirgen Therapeutics, which was founded with the mission of treating patients using RNA technology. Their c-srRNA, controllable self-replicating RNA, technology is being applied to telomere biology disorders where patients have inherited short telomeres and a failure to develop bone marrow. Elixirgen is looking to restore the ability to create blood cells in patients suffering from this rare disease.
Akihiro explains, "We are harnessing the tremendous potential of our controlled self-replicating RNA technology to redefine stem cell therapies for patients using the ZSCAN4 gene. We are working on potentially groundbreaking treatments for difficult-to-treat, rare, and genetic diseases, starting with the hematopoietic system and then going out into more indications."
"And our unique technology is that it is controllable in that it is optimized for expression at 30-35 ℃ and inactivated at temperatures at and above 37 ℃. And what that enables us to do is basically tune the expression of this self-replicating RNA. What is great is that self-replicating RNA has been known for its very long and also basically strong expression. However, it has been uncontrolled. So, we bring that control with this unique temperature-controlled system."
"So telomere biology disorders are a group of known diseases, and they are defined by these patients having inherited short telomeres and accelerated shortening of those telomeres over time. It is a rare disease, and the most common issue for the patients is that by the age of 30, around 80% of them develop bone marrow failure, which is a very serious condition."
"What this bone marrow failure entails is that their hematopoiesis, their ability to create new blood cells from their bone marrow, decreases over time. The reason for that is that the telomeres in their hematopoietic stem cells are shorter, and this reduces their ability to do so."
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