UMass Chan Medical School and Astellas Pharma Inc., a pharmaceutical company conducting business in more than 70 countries, have entered into a sponsored research agreement to proceed with research for an adeno-associated virus (AAV) vector mediated gene therapy for the treatment of Alexander disease, a fatal, ultra-rare disease for which the only treatment option is supportive care.
The research agreement supports research being conducted by Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research; and Jun Xie, PhD, associate professor of microbiology & physiological systems, who have developed an AAV vector that can express a small RNA silencer that suppresses the mutant proteins that cause Alexander disease in mice.
Dr. Gao and Dr. Xie will conduct additional research activities to accelerate their program toward clinical study, while Astellas will support UMass Chan investigations by providing drug discovery research expertise.
