Date: 2015-09-02
Type of information: Licensing agreement
Compound: therapeutics based on Sangamo’s zinc finger DNA-binding protein (ZFP) technology
Company: Shire (UK) Sangamo BioSciences (USA)
Therapeutic area: Hematological diseases - Genetic diseases
Type agreement: collaboration
R&D
licensing
Action mechanism: genome editing product. 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.
Disease: hemophilia A, hemophilia B, Huntington's disease and other monogenic diseases
Details: * On January 2012, Shire and Sangamo BioSciences have entered into 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 world-wide 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. Shire also receives the right to designate three additional gene targets. Using a mouse model of hemophilia B, Sangamo scientists and its collaborators have already established proof of concept that ZFN-mediated genome editing can be accomplished in vivo and is curative in the animal. They have demonstrated the production of stable levels of corrected human clotting Factor IX that are clinically meaningful, restoring clotting times to normal, after a single, systemic administration of ZFNs specific for the Factor IX gene. The data were published in Nature in June 2011 (Nature. 2011 Jun 26; 475(7355):217-21. doi: 10.1038/nature10177).
Financial terms: 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 is responsible for clinical development and commercialization of products arising from the alliance. Shire will pay Sangamo $13 million upfront followed by research, regulatory, development and commercial milestone payments, and royalties on product sales.
Latest news: * On September 2, 2015, Sangamo BioSciences announced that the company and its collaborator, Shire have agreed to revise their January 2012 collaboration and license agreement to expedite the development of ZFP Therapeutics for hemophilia A and B and Huntington's disease. The decision to restructure reflects a strategic decision by both Shire and Sangamo to focus efforts in areas of current interest and expertise for each company. Under the revised terms of the agreement, Shire will return to Sangamo the exclusive world-wide rights to gene targets for the development, clinical testing and commercialization of ZFP Therapeutics for hemophilia A and B. Shire will retain rights and will continue to develop ZFP Therapeutic clinical leads for Huntington's disease and a ZFP Therapeutic for one additional gene target yet to be named. Shire's rights with respect to other targets contemplated in the original agreement revert to Sangamo. Shire expects to file Investigational New Drug (IND) applications for the hemophilia B program and the first of our lysosomal storage disorder programs by the end of 2015. Under the revised agreement, each company is responsible for expenses associated with its own programs and will reimburse the other for any ongoing services provided. Sangamo has granted Shire a right of first negotiation to license the hemophilia A and B programs. No milestone payments will be made on any program and each company will pay certain royalties to the other on commercial sales up to a specified maximum cap. Additional financial details of the agreement will not be disclosed.
