Pfizer buys into gene therapy's renaissance and bets on Spark Therapeutics

Pfizer ($PFE) is the latest pharma heavyweight to get behind the promise of gene therapy, as the New York drugmaker has laid out plans to set up a dedicated R&D operation and signed a deal with startup Spark Therapeutics to kick-start the effort.

Michael Linden

The company has recruited Michael Linden, a professor at King's College London, to serve a two-year stint setting up a gene therapy research unit within Pfizer's existing rare disease segment. Linden, with more than 20 years of experience in gene therapy, will share his expertise in the field and help Pfizer's researchers get up to speed quickly, the company said, establishing a platform from which the drugmaker can build a pipeline of new treatments.

And Pfizer has found just the candidate to get things started in Spark's SPK-FIX. The treatment targets hemophilia B by using the harmless adeno-associated virus (AAV) to correct the errant gene responsible for the bleeding disease, potentially offering a one-shot cure. Spark, a 2014 Fierce 15 honoree, plans to take its treatment into Phase I/II development in the first half of next year, and, under the new deal, Pfizer will take responsibility for pivotal studies and regulatory submissions if and when they become necessary. Spark is due $20 million up front with up to $260 million more tied to developmental milestones.

Pfizer's interest in gene therapy comes amid something of a second honeymoon for a field that has been through the wash cycle of biotech hype. Serious safety issues and deliverability woes long hampered gene therapy R&D, but, after years of work, a new generation of academics and investigators believes it has hammered out the right viral vectors to safely and predictably get corrective genes to their target tissues, spurring renewed hope for widespread clinical success.

And now Pfizer wants in.

"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," Pfizer R&D Chief Mikael Dolsten said in a statement. "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 hematologic and neuromuscular diseases."

For Spark, Pfizer's co-sign spells another affirmation of the Philadelphia company's promise. Last month, the FDA conferred its coveted breakthrough therapy designation on SPK-RPE65, a Phase III treatment for inherited retinal dystrophies, and the promise of the biotech's pipeline has helped it raise more than $80 million in venture cash in under a year. The company spun out of the Children's Hospital of Philadelphia in 2013 with hopes of becoming a fully integrated pioneer in gene therapy, tapping the organization's expertise and recruiting a board of biopharma vets to light the way.

Pfizer's move is also the latest major affirmation of gene therapy's renewed luster among the world's largest drug developers. In September, Biogen Idec ($BIIB) made a splash in the field by unveiling plans to build an internal gene therapy division, one that will stand alongside its existing efforts in antisense therapies, biologics and traditional small molecules. Before that, Celgene ($CELG) teamed up with bluebird bio ($BLUE) on a gene therapy approach to oncology, and Bayer signed a $252 million deal with Fierce 15 designee Dimension Therapeutics to get its hands on a hemophilia A treatment. GlaxoSmithKline ($GSK), Baxter ($BAX) and others are working on in-house treatments in the field, as well.

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Special Report: FierceBiotech's 2014 Fierce 15 - Spark Therapeutics

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