Pfizer expands hemophilia gene therapy pipeline with $720m Sangamo deal

By Dan Stanton

- Last updated on GMT

Image: iStock/Perception7
Image: iStock/Perception7

Related tags Gene therapy

Pfizer has upped its presence in the gene therapy space through collaboration with Sangamo Therapeutics on its programmes worth up to $720m.

The agreement sees Pharma giant Pfizer pay Sangamo $70m upfront, but the Californian genome editing firm could receive up to $475m in milestone payments for its lead haemophilia A gene therapy SB-525, and a further $175m for other candidates developed under the collaboration.

Sangamo’s gene therapies are based on its multi-platform technology, including its zinc finger nuclease (ZFN) knock-out gene tool, and both adeno associated virus (AAV) and messenger RNA delivery tech.

“Sangamo brings deep scientific and technical expertise across multiple genomic platforms, and we look forward to working together to advance this potentially transformative treatment for patients living with Hemophilia A,”​ Pfizer’s president of Worldwide R&D Mikael Dolsten said in a statement.

The deal is the latest venture for Pfizer in the haemophilia gene therapy sector. The firm has a long-standing haemophilia B collaboration with Spark Therapuetics, inked in December 2014​ when Pfizer also established its gene therapy research unit.

Pfizer has since acquired Bamboo Therapeutics​ for $150m, adding an advanced recombinant AAV vector design and production technology and several pipeline therapies, and begun assessing sites in North Carolina​ to expand its gene therapy manufacturing capabilities.

“Pfizer has made significant investments in gene therapy over the last few years and we are building an industry-leading expertise in recombinant adeno-associated virus (rAAV) vector design and manufacturing,”​ said Dolsten.

“We believe SB-525 has the potential to be a best-in-class therapy that may provide patients with stable and durable levels of Factor VIII protein with a single administration treatment.”

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