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GEN News Highlights : Aug 27, 2013

Carolus, Alpha-1 Project Collaborate to Validate Inflammation-Related Disease Target

Carolus Therapeutics and The Alpha-1 Project (TAP), a subsidiary of the Alpha-1 Foundation, have entered a collaboration to characterize human biological samples for the presence of RANTES: Platelet Factor 4 heterodimer, a soluble protein complex that is secreted by activated platelets and implicated in many inflammation-related diseases, including genetic emphysema and COPD. TAP is making an equity investment in Carolus as a part of the partnership.

CT-2009, Carolus' lead compound, which selectively targets the heteromer, has been shown to be present and elevated in alpha-1 antitrypsin and COPD patient samples in preclinical studies, according to the company. The aim of the collaboration is to understand whether the concentration of the heteromer is related to the severity and progression of disease, and eventually whether heteromer levels can be successfully modulated by CT-2009 in patients.

In March, TAP made its first investment in a potential new treatment for alpha-1 antitrypsin deficiency, the most common known genetic risk factor for emphysema, by partially funding trials of a gene therapy treatment being developed by Applied Genetic Technologies.