It seems that gene technology is finally coming of age and there could be hope for persons suffering from diseases caused by genetic aberrations. With Pfizer Inc moving into the field of Gene therapy and this could herald a new revolution in the field of medicine. Technology to fix genetic aberration could be ready for trials after earlier setbacks.
The US drugmaker giant stated on Monday that it will be starting a gene therapy platform to study potential treatments for genetic disorders like hemophilia and sickle cell anemia. The genetic platform will be built with the help of a leading UK expert. Pfizer has also struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for hemophilia.
The Spark program is finally ready for early stage clinical trials for hemophilia B and will start in the first half of 2015. The Phase I/II tests will be conducted under the aegis of Spark while the later phase studies will be conducted by Pfizer. Pfizer will also be responsible for any regulatory approvals and potential commercialization.
Spark will get $20 million upfront and will also be eligible for extra payments based upon the success of the product in the market. The product is believed to have a market of $260 million.
Research effort in gene therapy by Pfizer will be led by professor from King’s College London and director of the University College London Gene Therapy Consortium, Michael Linden. Linden is joining Pfizer for a two year deputation.
Gene therapy is not a new concept and research has been continuing for the last 20 years. However research efforts were often dogged by repeated failures and also safety concerns. Today researchers have been able to solve some of the earlier problems and treatments have started reaching the clinics. The world’s first gene therapy is ready to go on sale in Germany to treat and ultra-rare blood disease.
Pfizer research head Mikael Dolsten said, “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.”