Cystic Fibrosis

Cystic Fibrosis

Lead program to treat patients with cystic fibrosis

Our modified polysaccharide drugs will improve the lives of patients with CF, by acting in the lungs and digestive tract to reduce abnormal mucus and biofilm adhesion, protect the mucosal barrier and suppress inflammation.

About cystic fibrosis

Cystic fibrosis (CF) is a life-threatening genetic disorder that results in decline of the lungs and digestive system. The accumulation of thick, sticky mucus clogs airways, leading to infection and chronic inflammation. Moreover, because of the inability to clear the airways, bacteria colonize and form biofilms that are difficult for antibiotics to penetrate. The mucus also causes blockage in the GI tract and inflammation similar to other inflammatory bowel diseases.

New treatments to change the progression of CF

Our lead program includes two drug candidates for treating the complications of cystic fibrosis. Synedgen is developing SYGN113 as an inhaled treatment to improve lung function of patients with CF. SYGN113 works by thinning and clearing thick mucus from the lungs. It also breaks up bacteria biofilms and suppresses inflammation.

Synedgen is developing SYGN303 as a potential treatment for distal intestinal obstructive syndrome (DIOS), in which thick mucus causes blockage in the intestine. DIOS is a GI complication associated with cystic fibrosis, for which there are no targeted or approved drugs.