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Biopharm Gains SBIR Grant for Cystic Fibrosis Treatment

Illustration of lungs (Mikael Häggström/Wikimedia Commons)

(Mikael Häggström/Wikimedia Commons)

OrPro Therapeutics Inc., a biopharmaceutical company in San Diego, received a Small Business Innovation Research (SBIR) grant from National Institutes of Health, to advance development of its lead product, ORP-100, for the treatment of cystic fibrosis. The company did not disclose the amount of the award from the National Heart, Lung and Blood Institute of NIH.

Cystic fibrosis is an inherited disease that causes thick, sticky mucus to build up in the lungs, digestive tract, and other areas of the body, and one of the most common chronic lung diseases in children and young adults. People who have cystic fibrosis inherit two faulty genes for the disease, one from each parent.

ORP-100 is an engineered variant of thioredoxin, a human lung protein that in laboratory studies has shown an ability to increase the fluidity of mucus. Clearance of the thickened mucus in the lungs that is characteristic of cystic fibrosis, and one that often remains poorly treated. ORP-100 will be administered through an aerosol delivery system, and targets the adhesive protein gel network common to patients with obstructive mucus.

ORP-100 is based on research conducted by pediatric pulmonologist Carl White at National Jewish Health hospital in Denver, Colorado.  In 2011, OrPro Therapeutics licensed from National Jewish Health the intellectual property for compositions and uses of thioredoxin to treat pulmonary diseases like cystic fibrosis.

The company says that despite advances in antibiotics and other treatments, there remains a large unmet need for improved mucus-reducing drugs which in many cases are the most effective way to relieve symptoms of the disease. OrPro says new treatments for cystic fibrosis are forecast to achieve peak annual sales exceeding $1 billion a year.

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