The University of Iowa (UI) Research Foundation recently announced it had struck a license and sponsored research agreement with Pfizer to support the development of a potential gene therapy for cystic fibrosis (CF). The research will be performed in the the laboratories of Profs. John Engelhardt and Ziying Yan.
Both Engelhardt and Yan are experienced gene therapy researchers who have been in the field for 25 years. The two groups will work together with Pfizer’s Genetic Medicines Institute, led by Michael Linden, to develop a new gene therapy for CF. Since the disease is caused by a mutation in a single gene, therapies aiming to restore gene function are attractive options when developing potential new treatments. Such therapies work by delivering a functional copy of the defective gene into the cells of a patient. The technique requires the use of a delivery system, usually in the form of a non-pathogenic virus.
The researchers will use an engineered hybrid of the adeno-associated virus (AVV) and the human bocavirus. The viral delivery system was developed by Engelhardt and Yan along with Prof. Jianming Qiu – a bocavirus expert from the University of Kansas. The viral vector also has the potential to be used for vaccine applications against lung viral infections.
In a press release, announcing the collaboration, Engelhardt said, “Efficiency of delivery is key to any gene therapy approach. The transport vehicle we have developed is an engineered virus that is highly efficient for entering human airway cells. Additionally, the ‘truck’ we use for transportation can carry a larger payload, which is especially important for cystic fibrosis since the diseased gene is very large.”
Now, the UI teams will test the efficacy of the viral delivery system in models of CF. Pfizer in turn will focus on developing packaging and optimizing the manufacturing process of the viral vector.
“This collaboration with Pfizer brings unique strengths that could enable the translation of this new vector system to clinical trials for CF lung disease and the discovery of potential new gene therapy applications for the vector system,” Engelhardt said.
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