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Enobia Pharma Presents PreClinical Data Showing ENB-0040 Significantly Improved Survival and Healed Skeletal Manifestations of Severe Hypophosphatasia in Mice
Date:9/15/2008

from Enobia Pharma will discuss the challenges in engineering the fusion molecule ENB-0040 consisting of TSNALP, an immunoglobulin Fc domain, and an anionic peptide used to target the enzyme to bone at the upcoming IBC 2008 Bioprocessing International Conference, Sept. 23-26, 2008, in Anaheim, CA.

About Hypophosphatasia

Hypophosphatasia is a rare, inherited, and sometimes fatal metabolic bone disease. Patients have low levels of the tissue non-specific form of alkaline phosphatase, an important regulator of bone mineralization, leading to rickets in infants and children and osteomalacia ("soft bones" resulting from poor mineralization) in adults. Disease severity is inversely proportional to the age at symptom onset, but morbidity can be cumulative and worsen with age. Clinical severity ranges from the severe perinatal or infantile form, with profound skeletal hypomineralization and respiratory compromise often causing death, to a more slowly progressive and debilitating osteomalacia in adults.

In the infantile form, infants may appear normal at birth but develop serious symptoms in the first six months of life. These can include failure to thrive, respiratory failure, fractures, and seizures. Radiographic findings include generalized hypomineralization and rickets. Mortality in these patients may be as high as 50%. In the childhood form, patients have varying degrees of hypomineralization, frank rickets, short stature, bone pain, muscle weakness, delayed motor milestones, early loss of deciduous teeth, and may experience frequent, poorly-healing fractures. In the adult form, the underlying osteomalacia causes bone pain due to overt or poorly-healing stress fractures that in some cases stops ambulation.

About ENB-0040

ENB-0040 is a fusion protein that includes the catalytic domain of human tissue non-specific alkaline phosphatase (TNSALP), an immunoglobulin Fc domain and a patented anionic peptide used to target the enzyme to bone
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SOURCE Enobia Pharma
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