ABHD18 ASO

ABHD18 ASO is an antisense oligonucleotide (ASO) in development by Scenic Biotech that targets abhydrolase domain-containing protein 18 (ABHD18), a gene identified as a novel modifier and missing link in the cardiolipin biosynthesis pathway. Cardiolipin is a phospholipid essential for mitochondrial function, and ABHD18 plays a key role in its remodeling and maturation by converting cardiolipin into monolysocardiolipin (MLCL). The drug's mechanism of action involves ASO-mediated suppression of ABHD18 expression (a mitochondrial modifier approach), which helps overcome the severe disease characteristics caused by the Barth syndrome-associated TAZ (tafazzin) gene mutation. The primary indication is cardiomyopathy, specifically in the context of Barth syndrome (BTHS), a rare inborn error of cardiolipin metabolism characterized by early-onset cardiac and skeletal myopathy.

Other names

ABHD18-targeted ASOABHD-18-targeted ASOABHD 18-targeted ASOMitochondrial modifier (ABHD18) ASO