TL-101

TL-101 is an investigational aerosolized recombinant adeno-associated virus (AAV) gene therapy candidate being developed by Talee Bio for the treatment of the pulmonary manifestations of cystic fibrosis. It is designed for inhaled delivery to the lungs to introduce a functional copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene into airway epithelial cells, with the goal of restoring CFTR activity and improving mucociliary clearance and lung function in patients regardless of their underlying CFTR mutation class.[9] As an early-stage gene therapy program, TL-101 is positioned as a potentially disease-modifying approach that could complement or provide an alternative to small-molecule CFTR modulators in cystic fibrosis.