Yesterday an 18-month-old research group called Rubius was officially launched with the mission to genetically engineer red blood cells for drug delivery and is targeting a number of diseases and conditions.
Rubius has as its first target the condition phenylkatonuria (PKU), a genetic disorder that prevents those with the disease from digesting the amino acid phenylalanine, which is found in most high-protein foods. The disorder can lead to severe problems and long-term health issues, including brain damage. Currently, the only treatment is a highly restrictive diet. The US sees about 300 new cases a year, and newborns are routinely tested for the disorder. Rubius's new treatment has been pre-clinically validated and is capable of enzymatically reducing phenylalanine levels in the human bloodstream.
The group claims to have developed more than 50 different therapies based on red blood cells, and predicts that the approach could lead to therapies for a wide range of conditions such as hematologic and autoimmune disorders, as well as infection diseases and cancer.
You can learn more about this new company and its work in its launch press release and in a report that appeared yesterday in Technology Review.