Binghamton pharmacy professor receives $1.9M award to tackle gene therapy challenges

Assistant Professor of Pharmaceutical Sciences Kuo-Ching (KC) Mei was thrilled to learn he and his team had won the $1.9M Maximizing Investigators' Research Award (MIRA) for Early Stage Investigators (ESI).

"My team and I have been very motivated by our work so it is truly an enabling award for Binghamton," shared Mei. "We have so many talented students and this award will enable them to do essential research for the next five years. It's empowering the research capacity that we have at the School of Pharmacy."

MIRA provides support for research in an investigator's laboratory that falls within the mission of the National Institute of General Medical Sciences (NIGMS). The goal of MIRA is to increase the efficiency of NIGMS funding by providing investigators with greater stability and flexibility, thereby enhancing scientific productivity and the chances for important breakthroughs.

Mei and his team are tackling a critical problem in gene therapy related to the immune system's response to treatment.

"We're working on ways to overcome the challenge of immunogenicity with Adeno-Associated Virus (AAV) vectors, which are commonly used in delivering gene therapy," explained Mei "Currently, if a patient has pre-existing immunity against AAV, they can't receive gene therapy at all. Even those who can, often have only one chance for treatment due to treatment-emerged immunity, and hoping it will be enough to cure their condition. Our goal is to find solutions that enable AAV gene delivery and allow more patients to safely receive and benefit from gene therapy."

Mei said this work is incredibly important because it could make gene therapy available to many more patients in need.

"Right now, some patients can't be treated because their immune systems would reject the therapy. For others, they have to rely on a single treatment to work perfectly as a one-and-done solution, which is a lot to ask," explained Mei. "By controlling the immune response to AAV, we can give these patients a better chance at successful treatment, offering new hope to those with genetic conditions that currently have limited or no treatment options."

School of Pharmacy and Pharmaceutical Sciences Dean Kanneboyina Nagaraju noted Mei's work has proven critical.

"Gene therapy is a viable therapeutic option for multiple genetic diseases," Nagaraju said. "One of the major hurdles is to overcome the immune response to vector and transgene upon redosing. Mei's work through this NIH grant has outstanding potential to address this important clinical problem. He is an exceptional scientist. I can't think of anyone better than him to address this practical problem in gene therapy."

The $1.9M award will support Mei and his team for the next five years.

"This grant will help us take our research to the next level by providing the resources we need to explore these critical questions," Mei explained. "It also means that students at our school will have the chance to be involved in this groundbreaking work. They'll learn firsthand how research can solve real problems, gaining valuable experience that will prepare them for careers in science and healthcare. This hands-on involvement in such important research will inspire and equip them to make their own contributions to the field in the future."

Mei concluded by acknowledging how this is a major milestone for his team: "It's an acknowledgment of the importance of our work and a vote of confidence in our ability to make a difference. Personally, it means a lot because it shows that the National Institute of Health recognizes and supports our efforts to improve gene therapy and delivery. This grant gives us the opportunity to keep pushing forward, find new ways to help patients, and contribute to the advancement of healthcare."

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