King’s College of London (KCL) has entered into an exclusive license agreement with Pfizer, Inc., for the development of a series of adeno-associated virus (AAV) gene therapy vectors. AAVs, a significant focus of many scientists in the field, act as delivery vehicles by carrying genetic material into living cells to achieve a sustained therapeutic effect without causing disease or triggering significant immune responses. The last several years have seen several advances in the development of gene therapy for rare diseases, including hemophilia A and B.
According to a KCL news release, the program was created using technology developed by Professor Michael Linden, PhD, and Dr. Els Henckaerts, a lecturer at KCL’s Division of Immunology, Infection & Inflammatory Disease. On December 1, 2015, Linden joined Pfizer from his position at KCL for a two-year “secondment” to lead gene therapy research within the company’s rare disease research area. A secondment provides an employee with a temporary transfer to another job for a specific time period for a specific purpose. Linden was a Professor of Virology on KCL’s Faculty of Life Sciences and Medicine, and Director of the University College London Gene Therapy Consortium.
Under the terms of the agreement, KCL will receive an upfront payment, and will be eligible for additional clinical development and commercialization milestone payments. In addition, as part of the Pfizer Rare Disease Consortium, Pfizer will fund research with Dr. Henckaerts for further development of the AAV vector platform and its application in gene therapy. It will also fund a new line of research in Henckaerts’ laboratory that is designed to apply insights into the basic understanding of the virus to help overcome the challenges of production for clinical use.
“The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients,” said Mikael Dolsten, MD, PhD, president of Worldwide Research and Development at Pfizer. “By establishing our gene therapy capabilities, we hope to gain a deeper understanding of the mechanisms that could potentially bring true disease modification for those suffering from devastating hematologic and neuromuscular diseases.”
Source: King’s College London news release dated January 19, 2016
