UMass Medical School has entered into a three-year, collaborative research agreement with Pfizer to evaluate determinants that influence the manufacturing quality and yield of viral vectors used in gene therapy. The research is being jointly conducted by Pfizer and UMMS, with the research at UMMS being performed under the direction of Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research, and Dan Wang, PhD, assistant professor of RNA therapeutics.
The UMMS three-year, collaborative research agreement with Pfizer will evaluate determinants that influence the manufacturing quality and yield of viral vectors used in gene therapy.
The work will focus on identifying cellular and viral variables that contribute to the production of high-quality recombinant adeno-associated viruses (rAAVs). Such rAAVs are the vectors used in certain in vivo gene therapies to deliver genetic information to cells.
AAVs are naturally occurring, nonpathogenic viruses that deliver their own genes into a host’s cells. By replacing the AAV genetic information with therapeutic genetic information (hence the recombinant form of the virus), genetic instructions that can interrupt or ameliorate a disease can be distributed to a patient’s cells. Typically, gene therapies either modify altered genes that produce disease-causing proteins or introduce a healthy copy of a gene if the altered gene results in a nonfunctional protein. An efficient process to robustly manufacture high quality rAAV vectors is an important element in developing effective gene therapy treatments.
“We know that many cellular and viral factors contribute to the quality of the viral vectors produced in the manufacturing process, but these factors have not been well characterized or optimized in the manufacturing process.” said Dr. Gao. “Working with Pfizer, we hope to illuminate some of these factors with the goal of optimizing the next generation of rAAV vectors for gene therapy.”
James P. McNamara, PhD, executive director of the Office of Technology Management at UMass Medical School, said UMMS is pleased to be working with Pfizer on “a key bottleneck in the gene therapy space.”
“Identifying those factors that are critical to manufacturing efficiency for gene therapy treatments will move the entire field forward,” said Dr. McNamara.
“Gene therapy holds great promise for patients with rare genetic disorders,” said John Ludwig, senior vice president and head of medicinal sciences at Pfizer. “Delivering these transformational therapies would benefit from significant improvements to manufacturing efficiency. With UMass Medical School, we’re ensuring our production utilizes leading edge biology to produce these potentially breakthrough medicines with high quality and efficiency."
At the Horae Gene Therapy Center, ongoing research is developing therapies for rare diseases such as Canavan, Tay-Sachs and Sandhoff diseases; retinitis pigmentosa; alpha-antitrypsin deficiency; cystic fibrosis; and Lou Gehrig’s and Huntington’s diseases.
