AAV8-TNAP-D10

AAV8-TNAP-D10 (also known as ARU-2801) is an investigational, preclinical-stage gene therapy designed as a one-time treatment for hypophosphatasia (HPP). Developed by Sanford Burnham Prebys Medical Discovery Institute and Nippon Medical School, with support from JCR Pharmaceuticals (and previously licensed by Aruvant Sciences), the therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a gene encoding a mineral-targeted form of tissue-nonspecific alkaline phosphatase (TNAP-D10). HPP is a rare genetic metabolic disorder caused by loss-of-function mutations in the ALPL gene, which leads to a deficiency in TNAP and the accumulation of inorganic pyrophosphate (PPi), a mineralization inhibitor. By continuously producing the missing TNAP enzyme, AAV8-TNAP-D10 aims to restore normal bone and dental mineralization and extend survival, offering a potentially curative alternative to lifelong, frequent subcutaneous injections of enzyme replacement therapy (such as asfotase alfa).

Other names

AAV8-TNALP-D10AAV-8-TNALP-D10AAV 8-TNALP-D10AAV8-TNAP-D10 gene therapyAAV-8-TNAP-D10 gene therapyAAV 8-TNAP-D10 gene therapyrecombinant AAV8-TNAP-D10recombinant AAV8-TNSALP-D10