Proteostasis will continue to focus its research and development on small molecules that correct defective CFTR protein in the cell and allow it to function properly.
In the Delta F508 mutation, the defective protein CFTR does not move to its proper place at the cell surface, impeding the flow of fluids into the airways. Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.
In addition to this collaboration, CFFT is investing in a robust pipeline of potential therapies that target CF from every angle, including research collaborations with Pfizer, Genzyme and Vertex to accelerate the discovery and development of new drugs.
CFFT's and the Foundation's goal is to find effective therapies and a cure for 100 percent of people with the disease.
Proteostasis Therapeutics develops disease-modifying therapeutics for orphan and neurodegenerative diseases.
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