Pfizer bets on gene therapy as technology comes of age

By Reuters

Dec 08, 2014 08:16 PM EST

Pfizer Inc is moving into the gene therapy space in the latest sign that the technology for fixing faulty genes may finally be ready for prime time, following earlier setbacks.

The U.S. drugmaker said on Monday it was establishing a gene therapy platform to study potential treatments, led by a top UK expert, and had struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for haemophilia.

The Spark program is expected to enter early-stage clinical trials for haemophilia B in the first half of 2015. Spark will be responsible for the early Phase I/II tests, with Pfizer taking over late-stage studies, any regulatory approvals and potential commercialization.

Spark will get $20 million upfront and be eligible for additional payments based on product success worth up to $260 million.

Pfizer's research effort in gene therapy will be led by Michael Linden, a professor from King's College London and director of the University College London Gene Therapy Consortium. Linden is joining Pfizer on a two-year secondment.

Gene therapy has seen more than 20 years of experiments but research has been dogged by a series of disappointments and safety concerns.

Now, however, scientists have solved some of the earlier problems and treatments are starting to reach the clinic, with a the Western world's first gene therapy set to go on sale in Germany to treat and ultra-rare blood disease.

"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," said Pfizer research head Mikael Dolsten.

Among other major pharmaceutical companies, Bayer AG struck a gene therapy deal with Dimension Therapeutics in June, while Novartis AG recently established a new cell and gene therapies unit, and Sanofi SA has a long-standing tie-up with Oxford BioMedica.

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