Pfizer to Intensify AAV Vector Research in Gene Therapy for Hemophilia

On December 8, Pfizer announced that it will establish a research program in gene therapy, and collaborate with Spark Therapeutics in Philadelphia, to develop potential gene therapy treatments for hemophilia.  The research would focus on utilizing bioengineered adeno-associated virus (AAV)-based vector, to develop therapies for hemophilia. Research in the field focuses on fostering expression of a clotting factor by administering a viral vector carrying that gene.

Under terms of the agreement, Spark will maintain responsibility for clinical development through Phase 1/2 studies. Pfizer will assume responsibility for pivotal studies, any regulatory approvals and potential global commercialization of the product.

Pfizer has also appointed Michael Linden, Ph.D., Professor at King’s College London and Director of the University College’s London Gene Therapy Consortium, to lead its research program in gene therapy for rare diseases.  Dr. Linden is an expert in AAV technology.

“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, M.D., Ph.D., 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.”

“The establishment of a gene therapy group under the leadership of Professor Linden will help Pfizer explore the potential of this important technology that could possibly benefit patients living with serious diseases,” said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “Professor Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field.”

Source: Pfizer