STK-002

STK-002 is a synthetic antisense oligonucleotide (ASO) developed by Stoke Therapeutics for the treatment of autosomal dominant optic atrophy (ADOA), a rare inherited optic neuropathy most frequently associated with mutations in the OPA1 gene. The drug is designed to prevent the inclusion of a naturally occurring nonproductive alternatively spliced exon in OPA1 pre-mRNA, which would otherwise lead to nonsense-mediated decay and reduced OPA1 protein levels. By blocking this alternative splicing event, STK-002 increases productive OPA1 mRNA and protein expression from the wild-type allele, aiming to restore mitochondrial function and slow or stop vision loss in ADOA patients. Preclinical studies have shown that intravitreal administration of STK-002 leads to increased OPA1 protein levels in retinal ganglion cells and improved mitochondrial respiration in patient-derived cells. The drug leverages Stoke’s TANGO (Targeted Augmentation of Nuclear Gene Output) platform technology and is currently being evaluated in Phase 1/2 clinical trials as the first potential disease-modifying therapy for ADOA[1][2][4][5][7][9].

Other names

SYNGAP1 ASOSYNGAP-1 ASOSYNGAP 1 ASOSTK-ASOStoke SYNGAP1 program