Apr. 28 at 9:09 AM
$CLLS ASGCT abstract 3414 In vivo targeted gene insertion using AAV and LNP formulated TALEN mRNA
drives phenylalanine lowering and metabolic rescue in a PKU model
1Alexion Pharmaceuticals, Cambridge, MA, United States,
2Cellectis, New York, NY, United States,
3Astrazeneca
4Cellectis, Paris, France,
5Alexion Pharmaceuticals, Boston, MA,
Phenylketonuria (PKU) is caused by inherited deficiency of phenylalanine hydroxylase (PAH),
most often due to missense PAH mutations, impairing conversion of phenylalanine to
tyrosine. Elevated phenylalanine in blood and brain causes neurotoxicity and developmental
impairment. Current standard of care including life-long dietary protein restriction and
approved therapies such as sapropterin and pegvaliase are either ineffective or carry black
box warnings, underscoring the need for new therapies.
