A breakthrough in gene therapy?
Reticulocytes will be forced to treat phenylketonuria
Vasily Sychev, N+1
The American company Rubius has developed a new technology for the treatment of certain diseases, which is supposed to use genetically modified red blood cells. According to the company, such red blood cells will be able to effectively treat many diseases. Rubius has already received $25 million from the "parent" Flagship Ventures to conduct clinical trials of a therapeutic method called Red-Cell Therapeutics. The development of the technology was carried out for 18 months.
According to the description of the technology on the company's website, modified reticulocytes, erythrocyte progenitor cells, will be used in the treatment.
Drawing from the website rubiustx.com – VMModification of such cells is supposed to be carried out at the stage of cultivation, after which reticulocytes become capable of producing biotherapeutic proteins.
Genetically modified blood cells can be used to treat metabolic disorders, autoimmune diseases and even cancer.
Other details of the therapeutic method are not disclosed. In general, Rubius has already developed treatment methods for 50 different diseases. Preliminary tests of the technology have already been carried out on animals, as well as human blood in laboratory conditions. Clinical trials are planned to begin with patients suffering from phenylketonuria, a hereditary disease associated with impaired metabolism of the amino acid phenylalanine.
Today, there is no clear therapy for the disease, and all methods are associated with a strict restriction of the intake of phenylalanine into the body with food. Rubius claims that their therapy will allow patients to abandon a strict diet and live almost a full life. Genetically modified reticulocytes will be responsible for removing phenylalanine from the bloodstream. According to the company, such blood cells are able to remain in the bloodstream for up to four months.
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10.12.2015