Biogen has announced a broad collaboration and alliance with the University of Pennsylvania (“Penn”) to advance gene therapy and gene editing technologies. The expansive research and translational development collaboration has multiple objectives, but will primarily focus on the development of therapeutic approaches that target the eye, skeletal muscle and the central nervous system (CNS). Another important aspect of the alliance will focus on validating next-generation gene transfer technology using adeno-associated virus (AAV) gene delivery vectors. The collaboration will also explore the expanded use of genome editing technology -- the insertion, deletion or replacement of DNA in the genome of an organism -- as a potential therapeutic platform.
This collaboration with Penn reinforces Biogen’s commitment to gene therapy and rare diseases. Biogen aims to build a robust gene transfer and gene editing platform for the future treatment of inherited or acquired diseases.
Biogen will work with James Wilson, M.D., Ph.D., Professor of Medicine and Pediatrics, and Director of Penn’s Gene Therapy Program, and Jean Bennett, M.D., Ph.D., Professor of Ophthalmology and Cell and Developmental Biology and Director of the Center for Advanced Retinal and Ocular Therapeutics. Both are recognized leaders in gene therapy in the Perelman School of Medicine at the University of Pennsylvania.
“We are committed to advancing gene therapy, and our collaboration with the University of Pennsylvania has significantly strengthened both our leadership and overall capabilities in the field,” said Olivier Danos, Ph.D., senior vice president, Cell & Gene Therapy at Biogen. “Joining forces with gene therapy pioneers, James Wilson and Jean Bennett, further enables Biogen to approach the technology from a powerful perspective. By exploring next-generation delivery in various tissues such as the retina, skeletal muscle and CNS, we will explore the potential for extending gene therapy beyond disorders linked to single gene mutations and into a broader spectrum of complex diseases, including devastating neurological conditions that affect a multitude of patients throughout the world.”
“My laboratory is committed to developing new technologies to address genetic diseases with few or no treatment options,” said Dr. Wilson, who is also the Director of the Orphan Disease Center at Penn. “This alliance is further evidence of Penn’s unflagging commitment towards enabling groundbreaking research and development in the cell and gene therapy field.”
“Recent advances in gene transfer and editing have now brought us to a promising stage and we believe that this exciting collaboration with Biogen will further fuel our collective ability to translate our research into viable gene therapies,” said Dr. Bennett.