Astellas Pharma US, Inc. has teamed up with UMass Chan Medical School to advance research on a groundbreaking gene therapy for Alexander disease, an ultra-rare and fatal condition. This innovative collaboration aims to bring new hope to patients through adeno-associated virus (AAV) vector-mediated therapy. The research, led by Dr. Jun Xie and Dr. Guangping Gao at UMass Chan, focuses on silencing the mutant proteins causing the disease. Together, we’re working to transform innovative science into life-changing treatments for those most in need. 💡🧬

We are thrilled to share this exciting news! 
Thank you to our many donors who helped End AxD support this promising gene therapy! Your gifts are making a difference!