rtx-134

RTX-134 is a genetically engineered allogeneic cellular therapy developed as a Red Cell Therapeutic product candidate. It consists of human red blood cells engineered to express the enzyme phenylalanine ammonia lyase (PAL) inside the cell. RTX-134 is designed to circulate in the bloodstream and degrade toxic levels of phenylalanine that accumulate due to a deficiency in the phenylalanine hydroxylase (PAH) enzyme, which causes phenylketonuria (PKU). By expressing PAL within red blood cells, RTX-134 aims for sustained reduction of serum phenylalanine with potentially fewer immune-driven adverse events compared to current therapies, as PAL is shielded from direct immune exposure by being encapsulated within the cell[1][5][6][8].