INZ-701 is a medication being studied for its safety and how well it is tolerated in infants. It is being tested in infants who have a deficiency in either ENPP1 or ABCC6, which are specific proteins in the body. The study aims to understand how the body processes this medication and its effects on these deficiencies.

ENPP1 Deficiency – ENPP1 Deficiency is a rare genetic disorder that affects the body’s ability to regulate calcium and phosphate levels. This condition can lead to abnormal calcification in various tissues, including blood vessels and joints. Over time, individuals may experience joint pain, stiffness, and potential cardiovascular complications due to the buildup of calcium deposits. The progression of symptoms can vary widely among affected individuals, with some experiencing more severe manifestations than others. The condition is typically inherited in an autosomal recessive pattern, meaning both copies of the gene in each cell have mutations.

ABCC6 Deficiency – ABCC6 Deficiency is a genetic disorder that primarily affects the skin, eyes, and cardiovascular system. It is characterized by the progressive calcification of elastic fibers in connective tissues, leading to symptoms such as skin lesions, vision problems, and cardiovascular issues. The disease often begins in childhood or early adulthood, with skin changes being one of the first noticeable signs. Over time, the calcification can lead to more severe complications, particularly affecting the heart and blood vessels. The condition is inherited in an autosomal recessive manner, requiring mutations in both copies of the ABCC6 gene.