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Pfizer establishes gene therapy unit for rare disease research
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  • Publication:2014/12/10

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.

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.

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.

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."