Dual Function Vector Platform
Our df-AAV platform allows treatment of other diseases with complex mechanisms where the mutant gene product must be reduced and the normal gene product augmented.
APB-101 is a "liver sparing" first-in-class gene therapy treatment for A1AT. It targets the liver via an AAV delivered dual function vector whereby the Z-AAT protein is silenced and M-AAT protein is augmented.
We are a spin-off from the University of Massachusetts Medical School (UMMS) and based upon nearly 30 years of gene therapy research of our scientific founders Dr. Chris Mueller and Dr. Terry Flotte.