AT982

**AT982** was an Audentes Therapeutics investigational gene-replacement candidate for Pompe disease. It is a recombinant adeno-associated virus serotype 8 vector carrying a human **GAA** expression cassette controlled by a novel hybrid promoter, designed to restore acid alpha-glucosidase activity in disease-relevant tissues, including skeletal muscle, cardiac muscle, the nervous system, and liver. Restored GAA activity is intended to reduce lysosomal glycogen accumulation and address limitations of enzyme-replacement therapy, including insufficient tissue penetration and immunogenicity. Audentes selected AT982 as its optimized Pompe clinical-development candidate in February 2018; the program was acquired by Astellas with Audentes. ([prnewswire.com](https://www.prnewswire.com/news-releases/audentes-therapeutics-announces-selection-of-optimized-clinical-development-candidate-for-pompe-disease-program-300594605.html))

Brand names

secGAA

Other names

AAV8-eMCK-hGAAAAV-8-eMCK-hGAAAAV 8-eMCK-hGAA