Beam Therapeutics Inc. has obtained IND clearance from the FDA for BEAM-304 for the treatment of phenylketonuria (PKU). BEAM-304 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct mutations in the phenylalanine hydroxylase (PAH) gene that cause PKU.
Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
Otsuka Pharmaceutical Co. Ltd. reaped positive results from an open-label phase II study of repinatrabit (JNT-517) in adolescents with phenylketonuria (PKU), a rare disease marked by the inability to break down the amino acid phenylalanine.
After two decades of work with Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. withdrew its bid for approval of the compound upon hearing from the U.S. FDA. Regulators told Warren, N.J.-based PTC that the data package for Translarna is “unlikely to meet the agency's threshold of substantial evidence of effectiveness.”
Phenylketonuria (PKU) is an autosomal recessive disorder that results in decreased metabolism of the amino acid phenylalanine. Untreated PKU can lead to intellectual disability, seizures, behavioral problems and mental disorders. This metabolic disease is caused by mutations in the phenylalanine hydroxylase (PAH) gene, resulting in patients’ inability to convert phenylalanine.
Genescience Pharmaceuticals Co. Ltd. has identified sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria (PKU).
Genescience Pharmaceuticals Co. Ltd. has identified phenylalanine hydroxylase (PAH) (R408W mutant) stabilizers reported to be useful for the treatment of phenylketonuria (PKU).
Maze Therapeutics Inc. CEO Jason Coloma said the latest phase I results with MZE-782, a prospect for phenylketonuria (PKU) as well as chronic kidney disease (CKD), bolster the firm’s “conviction to move into phase II” next year in both indications.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
