rAAV1-CBA-hAAT

rAAV1-CBA-hAAT is a gene therapy candidate designed for the treatment of alpha-1 antitrypsin deficiency (AATD). It utilizes a recombinant adeno-associated virus serotype 1 (rAAV1) vector to deliver a functional human alpha-1 antitrypsin (hAAT) gene, driven by a chicken beta-actin (CBA) promoter. The therapy is typically administered via intramuscular injection, targeting skeletal muscle cells to serve as a long-term biofactory for the production and secretion of the AAT protein into the bloodstream. This approach is intended to provide a steady supply of AAT to the lungs, where it inhibits neutrophil elastase and prevents the development of emphysema. The program has progressed through Phase 1 and Phase 2 clinical trials, with development involving the University of Massachusetts and Beacon Therapeutics.

Other names

recombinant adeno-associated virus alpha 1-antitrypsin gene vectorAAV1-CB-hAATAAV-1-CB-hAATAAV 1-CB-hAAT