THN-ASO1 (TFEB)

Theranexus a identifié un oligonucléotide antisens favorisant l’autophagie via une action directe sur TFEB.

TFEB, the major autophagy switch

Autophagy is a cellular process involving small intracellular vesicles called lysosomes and by which cells recycle part of their own material and also destroy certain residues which, by accumulating, can become toxic to them. This process, the discovery of which earned the Japanese Yoshinori Ohsumi the Nobel Prize in Medicine in 2016, is essential for proper cellular functioning, particularly that of neurons, and is impaired in a group of rare diseases called lysosomal diseases. Niemann Pick type C, Sanfilippo or Batten diseases are examples of lysosomal diseases. But beyond these rare diseases, autophagy is a process generally altered during aging and a deficiency in autophagy is today identified as being a determining element in diseases such as Parkinson's disease, Charcot's disease or age-related macular degeneration (AMD). TFEB is the protein recognized as being the molecular switch of autophagy, its activation is shown, in various in vitro or in vivo models, to present a strong therapeutic benefit in many pathologies.