ART001

ART001 refers to two distinct investigational drugs in current clinical development: - One is an orally available, selective phosphoinositide 3-kinase alpha (PI3Kα) inhibitor developed by ARTham Therapeutics for the treatment of slow-flow vascular malformations (SFVMs) and other PIK3CA-related overgrowth syndromes. This small molecule acts by inhibiting PI3Kα, a key enzyme involved in cell growth and angiogenesis pathways. Clinical studies have shown it to be effective and well-tolerated in both adult and pediatric populations[1][7][8][9]. - The other is a lipid nanoparticle (LNP)-delivered CRISPR-based in vivo gene editing therapy developed by AccurEdit Therapeutics for transthyretin amyloidosis (ATTR). This version of ART001 uses LNPs to deliver CRISPR components targeting the TTR gene directly into liver cells, resulting in durable reduction (>90%) of circulating TTR protein after a single infusion. It has received Orphan Drug Designation and RMAT designation from the FDA due to its promising safety profile—no infusion reactions or off-target edits observed—and potential as a one-time lifelong treatment for ATTR[2][3][4][5][6].