Path to a Cure: Many Routes, One Mission

The journey to end cystic fibrosis isn't a straight line. It is an evolving map with many paths and unique challenges. It requires an ambitious research agenda to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure.

1 min read

Explore the Path

The Foundation's Path to a Cure centers around three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF. Each approach requires a different set of scientific tools and knowledge, leading the Foundation to bring together researchers and industry leaders from a range of disciplines to advance multiple areas of research in parallel, driving progress toward our goal to make CF STAND FOR CURE FOUND.

The image shows the complicated path from the mutated CFTR gene to how we would repair, restore, or fix or replace the CFTR protein through CFTR modulators, nonsense-specific treatments, mRNA therapy, short nucleotide therapies, gene editing, or gene transfer until eventually we found a cure. Along the way, we have to figure out how to deliver genetic therapies by overcoming obstacles -- such as identifying the proper route and using the right vehicle -- and identifying the right cells to target.

Share this article

Topics

Research