Enzyme Replacement Therapy (ERT) to Restore Bone Mineralization
A subcutaneous enzyme replacement therapy with affinity for bone that is currently in clinical trials.ENB-0040 is an engineered A molecule that catalyzes or triggers biochemical reactions.enzyme developed by Enobia Pharma intended to treat A rare, inherited, and sometimes fatal metabolic bone disease characterized by poor bone mineralization and profound skeletal defects.hypophosphatasia (HPP). ENB-0040 is still under investigation.
HPP is caused by a deficiency in “tissue non-specific form” of alkaline phosphatase (TNSALP), an enzyme that plays a key role in regulating The process by which the body uses minerals to build bone structure.bone mineralization. Without TNSALP, Regulates certain intracellular functions and extracellular crystal formation.inorganic pyrophosphate (PPi) accumulates extracellulary and inhibits skeletal mineralization, leading to A softening of bones in children leading to fractures and deformity, which is predominantly caused by a vitamin D deficiency.rickets in pediatric patients or The adult form of rickets, which is a softening of the bones caused by defective bone mineralization.osteomalacia (soft bones) in adults.
ERTs work by reintroducing a missing enzyme directly to the diseased target tissue. In the case of ENB-0040, the therapy is proposed to directly target functional alkaline phosphatase (ALP) to the patient’s bones to help correct the enzyme deficiency which could lead to restoration of normal bone mineralization.