Cystic Fibrosis Market Snapshot: Disease-Modifying Drugs Elusive 24 Years After Discovery Of Root Cause
The discovery in 1989 of the underlying genetics causing cystic fibrosis was expected to spur broad development of disease-modifying therapies. As of 2013, however, only one such drug is on the market, for a small subset of CF patients, and a few other candidates are in clinical development.
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PTC Therapeutics looks to offer drug therapy for nonsense mutation CF patients, while Corbus is developing an anti-inflammatory, antifibrotic drug that it hopes will benefit the entire CF patient population.
The start-up asserts that agonizing the CB2 receptor will “resolve” inflammation, a different approach than used by anti-inflammatory drugs, and one that would pertain to the entire cystic fibrosis patient population.
A U.K. patient charity will fund preclinical studies of RPL554 for cystic fibrosis, Verona’s lead compound already in development for another niche respiratory condition, COPD exacerbations.