ADVM-053

ADVM-053 is a preclinical **gene therapy candidate** designed as a potential single-administration treatment for **hereditary angioedema (HAE)**. It uses an adeno-associated virus (AAV) vector (AAVrh.10) to deliver the gene encoding human C1 esterase inhibitor (C1EI), with the aim of inducing sustained therapeutic expression of C1EI protein. Deficiency in C1EI causes HAE, a rare genetic disorder characterized by unpredictable and potentially life-threatening swelling episodes. In preclinical models, a single intravenous dose of ADVM-053 increased C1EI protein expression above therapeutic levels and decreased vascular permeability, a relevant endpoint for HAE. This therapy is being developed by Adverum Biotechnologies and was granted Orphan Drug Designation by the FDA for hereditary angioedema. Development has since been discontinued before clinical trials due to strategic prioritization[1][2][3][5][7][9][12].

Other names

ADVM-053ADVM053ADVM 053AAVrh10-C1EIAAVrh-10-C1EIAAVrh 10-C1EIANN 002ANN002ANN-002AAVRH.10-C1EI