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Pfizer and Sangamo Therapeutics: Hemophilia A Gene Therapy

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Apr. 9 2018, Updated 12:41 p.m. ET

Hemophilia A

In May 2017, Sangamo Therapeutics (SGMO) and Pfizer (PFE) announced a collaboration for hemophilia A gene therapy that includes SB-525, which is one of Sangamo Therapeutics’ four lead product candidates. This collaboration is highly synergistic, as it combines Pfizer’s expertise in rare diseases with Sangamo Therapeutics’ expertise in genomic therapies.

In the last few years, Pfizer has invested significantly in gene therapy and is building industry-leading expertise in recombinant adeno-associated virus vector design and manufacturing. The company believes that SB-525 has the potential to be a best-in-class therapy that can provide patients with stable and durable levels of factor eight protein with a single administration treatment.

Under this collaboration agreement, Sangamo Therapeutics would receive $70.0 million from Pfizer in upfront payments. Sangamo Therapeutics would be responsible for conducting Phase 1 and Phase 2 clinical studies for SB-525 and certain manufacturing activities. 

Pfizer would be operationally and financially responsible for the subsequent research, development, manufacturing, and commercialization activities for SB-525.

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Potential payments

Sangamo Therapeutics (SGMO) is eligible to receive potential milestone payments of up to $475.0 million. These payments include up to $300.0 million for developing and commercializing SB-525 and up to $175.0 million for additional hemophilia A gene therapy product candidates that may be developed under the collaboration. Additionally, Sangamo Therapeutics would receive tiered double-digit royalties on net sales.

Hemophilia A is a rare blood disorder caused by genetic mutation. According to Pfizer, there are ~16,000 patients in the US and 150,00 worldwide with hemophilia A. Gene therapy treatments address the cause of diseases caused by genetic mutation. This technology involves introducing genetic material into the body to deliver a correct copy of a gene to a patient’s cells to compensate for a defective gene.

In the next part of this series, we’ll look at the licensing agreement between Pfizer and Basilea Pharmaceutica.

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