The Cystic Fibrosis Foundation (CFF) will fund the preclinical development of two gene therapy candidates, Talee Bio’s TL-101 and TL-102, for the treatment of cystic fibrosis (CF), the company announced.
Talee will receive up to $4.5 million from CFF to help accelerate the development of both these gene therapies for all CF patients, particularly for those who currently have no available therapies to treat the underlying cause of the disease.
CF is a genetic disease caused by mutations in the CFTR gene. Gene therapy aims to restore the function of the CFTR ion channel by delivering the correct copy of the gene into the cells of patients.
TL-101 and TL-102 are inhaled gene therapy candidates designed to help deliver the correct version of the CTFR gene through the use of a non-pathogenic virus. TL-101 is a recombinant adeno-associated virus (AAV)-based gene therapy, while TL-102 is a lentivirus-based gene therapy.
These therapeutic candidates have been developed after years of research to overcome certain challenges of gene therapy. Both Talee viral platforms are highly specific to human airway epithelia and, therefore, can “overcome the biggest limitation to using gene therapy to treat cystic fibrosis,” according to the company.
Both therapies were designed to treat the pulmonary symptoms of CF through either a single or several doses, in the case of TL-101, or only one dose, in the case of TL-102. At the moment, TL-101 is in preclinical development, and Talee hopes to start clinically testing the therapy in 2020.
All CF patients could be candidates for these two therapies, regardless of their specific CFTR mutation.
“Gene therapy treatments offer great potential to treat all patients with CF, including those with nonsense mutations and no available treatment options. We are honored to be working with the CF Foundation to expedite the development of these important development products in order to improve and extend the lives of every CF patient,” Lau said.