Gene Therapy, Pharma

Pfizer expands gene therapy effort on hemophilia, bagging rights to a Sangamo drug in a $545M deal

With one gene therapy for hemophilia B well along the way in the clinic at Spark Therapeutics, pharma giant Pfizer $PFE is now extending its interest in the field with a pact with Sangamo on a new gene therapy for hemophilia A.

The exclusive worldwide licensing deal on four programs at the longtime gene therapy biotech includes SB-525, which is poised on the threshold of a clinical trial.

Mikael Dolsten, Pfizer

“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,” noted Pfizer R&D chief Mikael Dolsten.

Sangamo is bringing its experience in the field to play, while Pfizer is once again whipping out a well used check book for the business development team. Pfizer is paying Sangamo $70 million upfront, up to $475 million in milestones — $300 million of which is devoted to SB-525.

Sangamo investors loved it, sending shares soaring 47% in after-market trading on Wednesday.

The deal puts Pfizer up against BioMarin $BMRN, which has been beaming recently over the data that it’s been seeing in hemophilia A. But Pfizer starts well behind BioMarin. Last summer the biotech said that it nailed down hard evidence that its gene therapy for hemophilia A demonstrated a high chance of restoring patients’ natural clotting abilities, reducing or eliminating bleeding episodes and pointing them down the road to a normal life. And company investigators pushed BMN 270 into a Phase IIb study aimed at gaining an accelerated approval for a potential blockbuster.

Sangamo says it is on track this quarter to start a Phase I/II clinical trial to evaluate safety and to measure blood levels of Factor VIII protein, along with other efficacy endpoints.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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