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December 20, 2024
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Binghamton pharmacy professor receives $1.9M award to tackle gene therapy challenges

Kuo-Ching Mei leads effort to tackle immunity barriers in delivering genetic medicine

Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy. Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy.
Assistant Professor of Pharmaceutical Sciences KC Mei standing in his lab at the School of Pharmacy. Image Credit: Scott Sasina.

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

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

MIRA provides support for research in an investigator鈥檚 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鈥檚 response to treatment.

鈥淲e鈥檙e working on ways to overcome the challenge of immunogenicity with Adeno-Associated Virus (AAV) vectors, which are commonly used in delivering gene therapy,鈥 explained Mei 鈥淐urrently, if a patient has pre-existing immunity against AAV, they can鈥檛 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.鈥

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

鈥淩ight now, some patients can鈥檛 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,鈥 explained Mei. 鈥淏y 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.鈥

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

鈥淕ene therapy is a viable therapeutic option for multiple genetic diseases,鈥 Nagaraju said. 鈥淥ne of the major hurdles is to overcome the immune response to vector and transgene upon redosing. Mei鈥檚 work through this NIH grant has outstanding potential to address this important clinical problem. He is an exceptional scientist. I can鈥檛 think of anyone better than him to address this practical problem in gene therapy.鈥

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

鈥淭his grant will help us take our research to the next level by providing the resources we need to explore these critical questions,鈥 Mei explained. 鈥淚t also means that students at our school will have the chance to be involved in this groundbreaking work. They鈥檒l 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.鈥

Mei concluded by acknowledging how this is a major milestone for his team: 鈥淚t鈥檚 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.鈥