Gene therapy for cystic fibrosis via inhalation has the potential to correct the CFTR defect and downstream pathophysiologic processes in the lung. All cystic fibrosis patients could be candidates for these treatments, regardless of the specific mutation. Talee is developing two inhaled gene therapy drug candidates to treat cystic fibrosis, TL-101 and TL-102. Both are designed to treat all cystic fibrosis patients, regardless of the specific CFTR mutation.
TL-101
- TL-101 is a recombinant AAV-based gene therapy to treat all forms of cystic fibrosis.
TL-102
- TL-102 is a lentiviral-based inhaled gene therapy to treat all forms of cystic fibrosis.