Enzyme Replacement Therapy (ERT) to Restore Bone Mineralization
ENB-0040 is an engineered enzyme developed by Enobia Pharma intended to treat 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 bone mineralization. Without TNSALP, inorganic pyrophosphate (PPi) accumulates extracellulary and inhibits skeletal mineralization, leading to rickets in pediatric patients or 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.