PRAX-090

PRAX-090 is an investigational antisense oligonucleotide (ASO) therapy being developed by Praxis Precision Medicines. It is designed to address SYNGAP1 loss-of-function (LoF) mutations, which are a leading cause of severe intellectual disability and epilepsy in developmental and epileptic encephalopathies (DEEs). The drug works by inhibiting the expression of synaptic Ras GTPase activating protein 1 (SYNGAP1), aiming to modulate gene expression through gene silencing mechanisms. As of mid-2025, PRAX-090 remains in preclinical development with no reported clinical trial data or regulatory approvals[1][2][3][4][5].