Message :
Required fields
BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today presented preclinical data suggesting the utility of its lead clinical development candidate, INZ-701, as a potential treatment for ABCC6 Deficiency. The data, presented at the virtual European Calcified Tissue Society Annual Congress (ECTS, May 6-8), are the first to show that an enzyme replacement therapy (ERT) increased plasma pyrophosphate (PPi) levels and reduced calcification in an animal model of ABCC6 Deficiency.
ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. This is one of several disorders with significant decrease in plasma PPi levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.