Phenylketonuria pipeline breaks into new modalities

With Synlogic picking a candidate to advance, the PKU field will soon have two programs in Phase III testing, and coming up behind them are at least 10 more programs spanning multiple therapeutic modalities that go beyond the indication’s approved small molecules and enzyme replacement therapies.

Phenylketonuria (PKU) is a rare, inherited metabolic disorder caused by mutations in the gene encoding PAH, an enzyme that normally converts phenylalanine to tyrosine. Mutations in the enzyme result in toxic buildup of the amino acid phenylalanine in the blood, which can affect the brain, leading to intellectual disability...