Endocrine Abstracts

Objectives: Hypophosphatasia (HPP) is an inborn error of metabolism resulting from loss of function mutations in the tissue-nonspecific alkaline phosphatase (TNAP) gene. Asfotase alfa is an approved enzyme replacement therapy for HPP, while effective, it requires chronic multiple injections up to 6 times per week. We have developed a one-time gene therapy drug (ARU-2801: an adeno-associated viral vector expressing TNAP-D10) for HPP and examined the safety and effica...