ToolGen partners with gene therapy company to develop special treatment for progeria

By Kim Joo-heon Posted : December 20, 2022, 17:29 Updated : January 6, 2023, 00:59

[Gettyimages Bank]

SEOUL -- ToolGen, a South Korean company which develops genome editing technology, partnered with ANL Bio, a domestic bio company specializing in gene therapy, to develop treatments for progeria, an extremely rare genetic disorder that causes rapid aging among young children. ToolGen's genetic scissors used to cut out specific genetic information in cells, will be combined with ANL's adeno-associated virus, a non-enveloped virus that can be engineered to deliver drugs to target cells.
Progeria, also known as Hutchinson-Gilford syndrome, causes children to age quickly starting at the age of two. The rare syndrome that takes place in one in every eight million people turns children's hair disappear along with their eyebrows and eyelashes. It is caused by the accumulation of a protein called "progeri" due to the mutation of genes involved in creating nuclear membranes in cell division. Zokinvy, a drug developed by a California-based biotech company called "Eiger" is the only progeria treatment approved by the U.S. Food and Drug Administration (FDA).
ToolGen said in a statement that ANL's adeno-associated virus (AAV) will be combined with its genetic scissors to develop a new treatment. Its efficacy will be evaluated through an animal model. ToolGen decided on the partnership as there are limitations to handling the disease only through its genetic editing technology. "With our outstanding partner, we will expand the pipeline by developing a treatment for progeria, which has been impossible to develop fundamental treatments," ToolGen CEO Kim Young-ho said in a statement on December 20. 
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