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Shire and Sangamo join to combat monogenic diseases

Wednesday, February 1, 2012 03:34 PM

Shire, a global specialty biopharmaceutical company, and Sangamo BioSciences, which offers genome-editing technology, have signed a collaboration and license agreement to develop therapeutics for hemophilia and other monogenic diseases based on Sangamo's zinc finger DNA-binding protein (ZFP) technology.

Shire will receive exclusive worldwide rights to ZFP Therapeutics designed to target four genes (for blood clotting factors VII, VIII, IX and X) which will be used to investigate curative therapies for hemophilia A and B—as well as the right to designate three additional gene targets.  Shire is responsible for clinical development and commercialization of products from the alliance. 

Sangamo is responsible for all activities through submission of Investigational New Drug (IND) Applications and European Clinical Trial Applications (CTA) for each product, and Shire will reimburse Sangamo for its internal and external research program-related costs. Shire will pay Sangamo $13 million upfront followed by research, regulatory, development and commercial milestone payments and royalties on product sales.

Sangamo's ZFP Therapeutic approach utilizes its proprietary ZFP nuclease (ZFN) and ZFP transcription factor (ZFP TF) technology. ZFPs can be engineered to recognize any specific DNA sequence within a gene, and may be applicable to certain Shire therapeutic areas, including hematology and lysosomal storage disorders.

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