Photo: Faith Ninivaggi
UMass Chan Medical School scientists Jun Xie, PhD, and Guangping Gao, PhD, have received $500,000 from Chengdu Origen Biotechnology Co. Ltd., a subsidiary of Chengdu Kanghong Pharmaceutical Group, to help advance gene therapy by improving gene delivery vehicle transduction efficiency.
Over the next 12 months, Dr. Xie, associate professor of genetic & cellular medicine, and Dr. Gao, the Penelope Booth Rockwell Chair in Biomedical Research, chair and professor of genetic & cellular medicine, director of the Horae Gene Therapy Center and director of the Li Weibo Institute for Rare Diseases Research, will focus on their research on improving AAV transduction efficiency..
“Improving AAV transduction efficiency allows for more effective targeting of tissues, especially those that are difficult to reach or specific cell types,” said Xie. “We’re excited to partner with Origen to develop a method to boost transduction efficiency, which will effectively reduce the required viral dose and mitigate the risk of immune responses in patients.”
Gene therapy holds transformative potential for treating a wide range of rare and inherited diseases. Using genetically modified viruses called viral vectors, —such as adeno-associated virus serotype (AAV)—, translational scientists can deliver genetic materials to cells with therapeutic benefits. However, AAV transduction efficiency in some organs or tissues remains suboptimal. Most notably, the immune system’s natural defenses against these viral vectors present a significant barrier to delivery.
To address this problem, the team will conduct a multi-phase research plan. In collaboration with Paul R. Thompson, PhD, the Endowed Chair in Biochemistry and Molecular Biotechnology II, professor of biochemistry & molecular biotechnology, and director of the Chemical Biology Program and the Small Molecule Screening Facility, the team will develop strategies to enhance AAV transduction efficiency through novel approaches in cell lines. Subsequently, the team will evaluate these strategies in animal models to observe their effect in vivo. If successful, this approach has the potential to expand the scope and durability of AAV-based gene therapies, benefiting a broader range of patients.
“We are proud to collaborate with UMass Chan Medical School, a leading institution in gene therapy research,” said Qiang Zheng, PhD, CEO of Chengdu Origen Biotechnology. “This partnership reflects our commitment to advancing scientific innovation and developing next-generation solutions that address key barriers in the field. We believe this research could pave the way for safer and more effective gene therapies worldwide.”
This partnership was facilitated by BRIDGE Innovation and Business Development at UMass Chan, which connects Medical School investigators with global partners to drive translational research and accelerate innovation with real-world impact.
