AdenoVerse GT

AdenoVerse GT is an engineered adenoviral gene therapy developed by Precigen for the treatment of alpha-1 antitrypsin deficiency (AATD). The vector is designed to address both the lung and liver manifestations of the disease by delivering a dual-action transgene cassette. This cassette provides sustained expression of the functional AAT PiM allele to protect the lungs from neutrophil elastase damage, while simultaneously utilizing a multi-miRNA module to silence the toxic, polymerized AAT PiZ allele in the liver. The vector utilizes a computationally-developed synthetic liver-specific promoter and a CpG-reduced transgene cassette to enhance potency, ensure liver-restricted expression, and minimize immune responses.