Full set in hemophilia

How Pfizer’s Sangamo deal fits into its hemophilia and gene therapy strategies

Pfizer Inc.’s deal with Sangamo Therapeutics Inc. brings in what the pharma believes is the last of three mechanisms it needs to cover the hemophilia population with new non-factor replacement therapies.

The companies announced the worldwide license and collaboration agreement to develop and commercialize gene therapies for hemophilia A on May 10. Sangamo expects to begin a Phase I/II study of lead program SB-525 this month.

Sangamo will receive $70 million up front and is eligible for up to $300 million in milestones for SB-525, and up to $175 million for additional products developed in the collaboration. The biotech may also receive tiered, double-digit royalties.

Pfizer has a gene therapy for hemophilia B in Phase I/II testing and a bypass agent for hemophilia A or B in Phase II.

The hope is the gene therapies would be curative, but even it they aren’t, they could dramatically change patients’ quality of life by

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