Pfizer Inc.  announced two strategic decisions to expand the company’s rare disease research and development activities through the establishment of a gene therapy platform to investigate potential treatments for patients. First is an agreement with Spark Therapeutics to develop SPK-FIX, a programme incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B expected to enter phase 1/2 clinical trials in the first half of 2015.

Additionally, Pfizer has appointed Michael Linden, Ph.D., Professor at King’s College London and Director of the University College London Gene Therapy Consortium, who will be with the company for a two-year secondment to lead gene therapy research in the rare disease area.

“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.”

Philadelphia-based Spark Therapeutics and Pfizer will collaborate to progress the clinical programme for SPK-FIX, a programme incorporating a bio-engineered AAV vector for the potential treatment of hemophilia B. Pfizer has a long-standing commitment to the hemophilia community and has been providing hemophilia products to patients for more than 17 years.

“Pfizer strives to provide meaningful enhancements to the lives of patients with hemophilia, and the agreement with Spark Therapeutics offers an important expansion of Pfizer’s commitment to the bleeding disorder community and builds on our leading hemophilia portfolio,” said Geno Germano, group president, Global Innovative Pharma Business at Pfizer. “We believe the SPK-FIX programme could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for patients with bleeding disorders.”

Under the 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.

Effective December 1, 2014, Professor Michael Linden has joined Pfizer from his current position at King’s College London, for a two-year secondment to lead gene therapy research within the company’s rare disease research area.

“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.”

Rare diseases are among the most serious of all illnesses and impact millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact in addressing unmet medical needs. The Pfizer focus on rare diseases builds on more than a decade of experience and a global portfolio of 22 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.