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Horae Gene Therapy Center now Department of Genetic & Cellular Medicine

by Alexandra Hartman Editor-in-Chief

Gene Therapy Pioneer Announces Creation of Academic Department at UMass Chan

Guangping Gao, PhD, is founding chair of the Department of Genetic & Cellular Medicine.
Photo: Faith Ninivaggi

The field of gene and cell therapy has taken a giant leap forward at UMass Chan Medical School with the establishment of a brand-new academic department.

The Horae Gene Therapy Center, a pioneer in its field since 2007, has transitioned into the Department of Genetic & Cellular Medicine, solidifying UMass Chan’s position as a global leader in advancing therapeutic solutions for rare diseases. Leading this pioneering effort is Guangping Gao, PhD, now the founding chair of the new department.

Dr. Gao, the Penelope Booth Rockwell Professor in Biomedical Research and professor of microbiology & physiological systems, expresses enthusiasm about the new venture: “This is a very exciting moment for our gene therapy center. We are no longer simply a research center. This new status makes us the first gene and cell therapy academic department in the country.”

Breaking New Ground in Rare Disease Treatment

The department will continue to leverage the gained expertise and success of the Horae Gene Therapy Center. Established over 15 years ago, the center has been instrumental in developing transformative treatments for rare inherited diseases lacking existing cures, including conditions like cystic fibrosis, Huntington’s disease, and amyotrophic lateral sclerosis (ALS).

Under Dr. Gao’s leadership, the center’s research has also been crucial in laying the groundwork for some of the first clinical trials dedicated to novel therapeutics for neurodegenerative disorders such as Tay-Sachs, ALS, and Canavan disease. The team’s remarkable achievements have been fueled by the development of various AAV vectors—small, single-stranded DNA viruses repurposed to deliver gene therapy medicines.

Driven by their groundbreaking research, the center has attracted significant funding, securing tens of millions of dollars from the National Institutes of Health and fostering remarkable relationships with both industry partners and non-profit organizations, securing millions more in funding support.

Expanding Horizons with a Multifaceteded Approach

The transition to an academic department opens up a world of opportunities for Dr. Gao and his dedicated team.

With this new status they are poised to expand their research program, nurture a new generation of researchers, and further boost collaboration among faculty members committed to rewriting the narrative for those battling rare diseases.

“We are indeed among the leaders in gene and cell therapy in the country and around the world, and this transition to an academic department will only further distinguish the work being done here at UMass Chan,” said Dr. Gao.

Expanding these capabilities aligns perfectly with the department’s commitment to educating future leaders. The new department is prepared to pursue NIH training grants such as the T32 program, offering valuable fellowships to postdoctoral trainees—the innovators who will usher in a new era of gene therapies.

Innovators Pitching In: From Discovery to Translational Research

Joining Dr. Gao’s visionary team is Negaunee

Jennifer adjourned to

Adair, PhD

laboratory is committed

Jennifer Adair, PhD, brings her expertise as she assumes the role of associate director of the department and vice chair of the Department of Genetic & Cellular Medicine.

Dr. Adair is a globally recognized leader in genetically modified cell therapies targeting genetic, malicious, and infectious diseases. She joins UMass Chan from the renowned Fred Hutch Cancer Center and the University of Washington.

Adding to the department’s scientific prowess, Dr. Adair will spearhead the launch of the

Ex Vivo Gene Therapy and Editing Core, where

a new asset

She will also spearhead the development of pioneering lentiviral and non-viral platforms as a testament to the department’s commitment to advancing research and providing patients with more options.
Dr. Adair will officially begin her role in spring 2025.

Her

director

with the center’s bibliography
expertise in in vivo approaches

This department markes a significant moment for UMass Chan, establishing the

an academic institution, simlar to that of its peers, solidifies teu position as a EEuan Medical School.

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