Be a part of a new era in cellular medicine for the potential treatment of phenylketonuria
PKU is an inherited metabolic disorder that is characterized by the body’s inability to metabolize the essential dietary amino acid, phenylalanine, due to a lack of or deficiency in the enzyme phenylalanine hydroxylase (PAH).
Without PAH, phenylalanine can build up to dangerous levels in a person’s body and lead to severe cognitive dysfunction if left untreated.
Rubius Therapeutics is developing a potential new treatment option for phenylketonuria (PKU), called RTX-134. RTX-134 is an enzyme replacement therapy with the enzyme phenylalanine ammonia lyase (PAL) inside a red blood cell. PAL is used as a substitute for the missing PAH enzyme with the goal of breaking down phenylalanine as it circulates through the bloodstream.
At Rubius Therapeutics, our ultimate goal in developing RTX-134 is to provide patients with a safe and effective treatment option that lowers phenylalanine via infrequent administration and allows people to enjoy a normal diet.