ZFP technology forms basis of $13 million Sangamo, Shire agreement

RICHMOND, Calif.—Sangamo BioSciences, Inc. and globalspecialty biopharmaceutical company Shire plc have announced a newcollaboration and license agreement, under which the companies will developtherapeutics for hemophilia and other monogenic diseases, based on Sangamo'szinc finger DNA-binding protein (ZFP) technology.

"We are delighted to be partnering the first of ourmonogenic disease programs with Shire, a company known for its development ofinnovative medicines for genetic diseases," Edward Lanphier, Sangamo'sPresident and Chief Executive Officer, said in a press release. "This allianceis further validation of our ZFP platform as a transformative technology forthe development of novel therapeutics, which have the potential torevolutionize the treatment of a wide range of genetic diseases."

Under the terms of the agreement, Shire will be grantedexclusive worldwide rights to ZFP Therapeutics designed to target fourgenes—for blood clotting Factors VII, VIII, IX and X—which will be used toexplore curative therapies for hemophilia A and B. in addition, Shire will alsohave the right to designate three additional gene targets. Sangamo will receive$13 million up front, as well as research, regulatory, development andcommercial milestones, and will be responsible for all activities up throughthe filing of IND applications and European Clinical Trial applications foreach project. Shire will reimburse Sangamo for its internal and externalresearch program-related costs, and will be responsible for clinicaldevelopment and commercialization of products that result from the agreement.

The ZFP Therapeutic approach is based on Sangamo'sproprietary ZFP nuclease and ZFP transcription factor technology, and ZFPs canbe engineered to recognize any specific DNA sequence within a gene. Thetranscription factors provide the ability to turn genes on or off, while zincfinger nucleases (ZFNs) are capable of enabling gene editing within cells. TheZFNs make it possible to modify the DNA of a cell at a precise location,allowing for the correction or disruption of a specific gene or the addition ofa new DNA sequence.

"Sangamo's ground-breaking ZFP gene-editing technology willenable us to expand our therapeutic pipeline into therapies for other geneticdisorders such as hemophilia," Sylvie Gregoire, president of Shire's HumanGenetic Therapies business, said in a press release. "While still early in theclinical development process, this DNA-binding protein technology is alignedwith our focus of developing new treatments that can add value for physicians,patients and their families, and the healthcare community overall."

SOURCE: Sangamo BioSciences, Inc. press release