Following a series of past setbacks in the gene therapy space, Pfizer Incorporated has partnered with another US biotech firm, Spark Therapeutics, to develop effective gene therapy technology and a treatment for hemophilia – a congenital tendency to uncontrolled bleeding, usually affecting males and transmitted from mother to son.

A top UK gene therapy expert, Michael Linden has been contracted to lead Pfizer’s gene therapy research effort on a two-year secondment, and hopes of success are high considering the credentials of the persons and companies involved in this breakthrough effort. Linden is a professor from King’s College London, and director of the University College London Gene Therapy Consortium.

This is not the first time pharmaceutical companies have been making effort to succeed at gene therapy research in the past 20 years, but safety concerns and technical disappointments have been dogging the efforts and stemming the possibilities of success.

According to the research head for Pfizer, Mikael Dolsten, “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.”

As part of its partnership with Spark Therapeutics, Pfizer will provide Spark with $20 million upfront payment and based on product success and acceptability, Spark will be eligible for additional funds of up to $260 million. The biotech firm will also commence on first clinical trials by early 2015 for hemophilia B, and Pfizer will handle last-stage trials, including FDA approvals and marketing functions for the product.

The world’s first gene therapy drug is already on sale in Germany for $1.4 million; and other pharmaceutical firms are already striking partnerships to succeed at the effort – among these are Bayer AG entering into a deal for gene therapy with Dimension Therapeutics, Sanofi SA tying up a deal with Oxford BioMedica, and Novartis AG already setting up a cell and gene therapy units.

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