Hemophilia B, a rare bleeding disorder, affects about 26,000 males worldwide. To control bleeding episodes, individuals who have this genetic disorder are treated through repeated intravenous infusion of recombinant or plasma-derived Factor IX, which play a vital role in blood coagulation.
A new gene therapy to address this genetic condition, however, may soon be available with drug company Pfizer Inc. announcing on Monday that it is establishing a gene therapy platform to study prospective treatments for patients with hemophilia.
"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," said Pfizer Worldwide Research and Development President Mikael Dolsten.
The pharmaceutical company is also collaborating with biotech company Spark Therapeutics for the development of a treatment for the disease via the latter's SPK-FIX program which incorporates a bio-engineered adeno-associated virus (AAV) vector that delivers a high-activity Factor IX gene to the patient's liver as a potential treatment for the hematologic disorder.
"With their experience in the field of gene therapy, as well as in the research and development of potential novel treatments for hemophilia, we believe that Spark's team of scientists and clinicians will complement Pfizer's expertise in helping to bring a new therapy to patients," said Pfizer's Rare Disease Research Unit chief scientific officer Kevin Lee.
Under the terms of their agreement, Spark will get an upfront payment of $20 million from Pfizer, and is eligible to receive another $260 million depending on the success of the product.
Early-stage clinical trials of the hemophilia B Spark program is set to start in the first half of 2015. The gene-therapy company will take charge of the early Phase I/II tests but Pfizer will take over the late-stage studies. It will also be responsible for regulatory approvals and product's potential commercialization.
Although researchers have been experimenting on gene therapy for more than 20 years, these involved a series of disappointments and safety concerns, but now that scientists were able to solve some the problems they have earlier encountered with the treatment, pharmaceutical companies now see opportunity in gene therapy. Besides Pfizer, other drug companies that are investing on gene therapy include Bayer, Novartis and Sanofi.
"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," Dolsten said.