Pfizer

US-based drugmaker Pfizer has established a gene therapy platform to investigate potential treatments for patients.

The company aims to expand its rare disease research and development activities.

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Pfizer worldwide research and development president Mikael Dolsten said: "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 haematologic and neuromuscular diseases."

Pfizer signed an agreement with Philadelphia-based Spark Therapeutics to develop SPK-FIX, a programme incorporating a bio-engineered AAV vector for the potential treatment of haemophilia B.

"Spark will maintain responsibility for clinical development through Phase I/II studies, while Pfizer will take responsibility for pivotal studies, regulatory approvals and potential global commercialisation of the product."

It is expected to enter Phase I/II clinical trials in the first half of 2015, according to Pfizer.

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Under the terms of the agreement, Spark will maintain responsibility for clinical development through Phase I/II studies, while Pfizer will take responsibility for pivotal studies, regulatory approvals and potential global commercialisation of the product.

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Pfizer appointed Michael Linden, professor at King’s College London and director of University College London Gene Therapy Consortium, for a two-year secondment to lead gene therapy research within the company’s rare disease research area.

Pfizer rare disease research unit senior vice-president and chief scientific officer Kevin Lee said: "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."

Image: Pfizer World Headquarters. Photo: courtesy of Norbert Nagel, Mörfelden-Walldorf, Germany.