Pfizer, Sangamo Partner in Gene Therapy in Deal Worth Up to $545 Million

Pfizer has formed an exclusive, global collaboration and license agreement, worth up to $545 million, with Richmond, California-based Sangamo Therapeutics, for the development and commercialization of gene-therapy programs for hemophilia A, including SB-525, one of Sangamo’s four lead product candidates, which Sangamo expects will enter the clinic this quarter.

Under the agreement, Sangamo will receive a $70-million upfront payment from Pfizer and will be responsible for conducting the SB-525 Phase I/II clinical study and certain manufacturing activities. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing, and commercialization activities for SB-525 and any additional products. Sangamo is eligible to receive potential milestone payments of up to $475 million, including up to $300 million for the development and commercialization of SB-525 and up to $175 million for additional hemophilia A gene-therapy product candidates that may be developed under the collaboration. Sangamo will also receive tiered double-digit royalties on net sales. Additionally, Sangamo will be collaborating with Pfizer on manufacturing and technical operations using viral delivery vectors.

Pfizer has been building its capabilities in gene therapy, including for viral product manufacturing. In 2016, Pfizer acquired Bamboo Therapeutics, a privately held biotechnology company based in Chapel Hill, North Carolina, focused on developing gene therapies. The acquisition provided Pfizer with a clinical and several preclinical assets, recombinant adeno-associated virus (rAAV) vector design and production technology, and a Phase I/II gene-therapy manufacturing facility that Bamboo had earlier acquired from the University of North Carolina.

Also in 2016, Pfizer and its collaboration partner, Spark Therapeutics, a Philadelphia, Pennsylvania-based gene therapy company, received breakthrough therapy designation by the US Food and Drug Administration for SPK-9001, the companies’ lead investigational candidate for treating hemophilia B. The compound is a bio-engineered AAV capsid that is in an ongoing Phase I/II trial. Breakthrough therapy designation is intended to expedite development and FDA review. The companies had formed a collaboration in gene therapy in 2014.

Source: Pfizer


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