rAAV9.EF1a.PLA2G6

rAAV9.EF1a.PLA2G6 is an adeno-associated virus serotype 9 (AAV9) gene therapy candidate designed for the treatment of infantile neuroaxonal dystrophy (INAD). INAD is an ultra-rare, autosomal recessive neurodegenerative disorder caused by mutations in the *PLA2G6* gene, which encodes a calcium-independent phospholipase A2 enzyme essential for phospholipid metabolism and mitochondrial function. The therapy utilizes the EF1a promoter to drive the expression of a functional human *PLA2G6* transgene. It is administered via intra-cisterna magna (ICM) injection to achieve widespread distribution throughout the central nervous system. Developed by researchers at Nationwide Children's Hospital with support from the INADcure Foundation, the program has completed preclinical toxicology studies in rats to support a planned Investigational New Drug (IND) application.