Developing a new triple combination therapy
Cystic fibrosis is the most common genetic disorder affecting approximately 70,000 to 100,000 people worldwide. Improvement in disease management protocols and approval of new drugs to treat the symptoms have extended the life expectancy for CF patients and is now approaching 40 years of age. However, CF remains an incurable disease that leads to death. Recently, CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) mutation-specific disease modulators such as correctors and potentiators, have shown the ability to restore CFTR function in selected genotypes.
We exploit our proprietary Disease-Relevant Translation, DRT™ platform which utilizes functionally pertinent phenotypic assays and disease relevant models to identify hit molecules that act on the protein homeostasis pathways within the cell and elicit higher CFTR mediated chloride currents
This program is fully owned by Proteostasis Therapeutics.