LX1004

LX1004 is an AAV-mediated gene therapy being developed for the treatment of **CLN2 Batten disease** (late-infantile neuronal ceroid lipofuscinosis). The therapy uses an adeno-associated virus (AAV) vector to deliver a fully functional CLN2 gene directly to the central nervous system (CNS) via intracisternal injection. This gene encodes tripeptidyl peptidase 1 (TPP1), a protein deficient in patients with CLN2 disease, to restore its normal function. CLN2 Batten disease is a fatal lysosomal storage disorder of childhood caused by CLN2 mutation, resulting in progressive neurodegeneration. LX1004 has demonstrated a favorable safety profile and statistically significant reduction in disease progression rate in a completed phase 1/2 trial. The drug has received Orphan Drug and Rare Pediatric Disease designations from US and European regulators[1][3][4][6][7][9][10].