The Cystic Fibrosis Foundation awarded up to $4.5 million to Talee Bio Inc. to advance novel treatments that could benefit all individuals with CF regardless of their underlying mutation. The funds will be used to identify new mechanisms to deliver a healthy cystic fibrosis transmembrane conductance regulator (CFTR) gene to the lungs, a critical step in developing gene therapy for the disease.
Successful delivery of the CFTR gene -- which has been the focus of substantial research since the gene's discovery -- has been a significant hurdle in the development of gene therapy for CF because the body's natural defenses fight to keep anything foreign out of the lungs (such as bacteria and viruses). Researchers believe that introducing a healthy gene into airway cells could result in the production of normal CFTR proteins in the lungs of all people with CF, including individuals with rare and nonsense mutations who do not have treatments for the underlying cause of the disease.
The $4.5 million is the total of two awards from the Foundation to Talee Bio to advance their gene delivery program. The company is developing two gene delivery candidates with the goal of starting early stage clinical trials with one of them as soon as 2020.
The Foundation is steadfast in its dedication to finding treatments -- and ultimately a cure -- for the underlying cause of the disease for all people with CF, including those with nonsense and rare mutations who will not benefit from CFTR modulators. It has committed more than $90 million from 2018 onwards for the development of new treatments for these individuals, including gene therapy that would benefit everyone with CF regardless of their mutation.
To learn more about the research the Foundation is supporting, please visit the Research We Fund section of cff.org.