Cystic Fibrosis Gene Therapy to Benefit from Collaboration Between Pfizer, University of Iowa

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.