THN-ASO1

THN-ASO1 is an antisense oligonucleotide (ASO) developed by Theranexus (operating as THX Pharma) in collaboration with Inserm and Diverchim through the PickASO project. It is designed to target Transcription Factor EB (TFEB), a master regulator of the autophagy-lysosomal pathway. By directly acting on TFEB, the drug aims to promote autophagy and lysosomal biogenesis, facilitating the clearance of cellular waste and toxic protein aggregates. THN-ASO1 is currently in preclinical development for the treatment of various lysosomal storage disorders, including Niemann-Pick type C, Sanfilippo disease, and Batten disease. It also holds therapeutic potential for broader neurodegenerative and age-related conditions such as Parkinson's disease, amyotrophic lateral sclerosis (ALS), and age-related macular degeneration.