Despite the number of mixed results in gene therapy which has had its share of setbacks, drug company giant Pfizer Inc. is pushing through with its gene therapy project. This is backed by advancement in technology and better research facilities, and for its first project, a partnership with privately-owned biotech firm Spark Therapeutics.
Pfizer said it was establishing a gene therapy platform to study potential treatment for hemophilia, among other diseases in a recent press release distributed by the company. The alliance with Spark is seen to hasten the development of the prospective treatment by incorporating a bio-engineered AAV vector for Hemophilia B treatment.
Mikael Dolsten, M.D., Ph.D, president of Worldwide Research and Development with Pfizer says, "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. 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."
Reports said that Spark would get $20 million upfront and be eligible for additional payments based on product success worth up to $260 million.
The program is expected to enter early-stage clinical trials for haemophilia B in the first half of 2015.
Pfizer group president of Global Innovative Pharma Business, Geno Germano, said, "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. We believe the SPK-FIX program could add to our existing portfolio of hemophilia products and could pioneer a potential new treatment technology for our patients with bleeding disorders."
Pfizer's research effort in gene therapy will be led by Michael Linden, a professor from King's College London and director of the University College London Gene Therapy Consortium.
Senior vice president and chief scientific officer of Pfizer's Rare Disease Research Unit, Kevin Lee, Ph.D, says "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. Professor Linden brings to Pfizer his extensive expertise in AAV technology obtained from over 20 years working in the field."
