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treat the specific tissues where destructive inflammation occurs, Celniker says. Blood transfusions are a standard treatment for aHUS, a blood disease that causes blood clots in the kidneys due to inflammation. The transfusions temporarily replenish patients with healthy blood cells, but Taligen’s protein drug aims to prevent the aberrant complement system activities that cause damage to the blood cells in the first place.
For patients with PNH, who also get blood transfusions, cortico steroids have also been used to suppress immune activities that cause the destruction of red blood cells. The downside is that reducing the body’s immune activities broadly like steroids do can hurt the body’s ability to fight off infections. Taligen’s big competition in the PNH market would be Cheshire, CT-based Alexion Pharmaceuticals (NASDAQ:ALXN), which makes an antibody drug called eculizumab (Soliris) that protects red blood cells from the immune system attacks that the disease causes. Yet Celniker says that a potential advantage of her firm’s drugs is better control over the destruction of red blood cells.
Taligen expects to begin Phase I clinical studies of the drug for the rare diseases before the end of the year, Celniker says. The company wants to find pharmaceutical partners to develop its drug for disease that affect larger groups of patients such as age-related macular degeneration, she said. Since we last talked to the firm back in 2008, it closed its R&D operation in Colorado, and consolidated its labs and offices mostly under one roof in Cambridge. The firm’s chief scientist and co-founder, Michael Holers, still has an office in Denver, Celniker said.