Date: 2016-11-30
Type of information: Licensing agreement
Compound: zinc finger nuclease (ZFN)-mediated genome editing program
Company: Bioverativ (USA - MA) Sangamo Biosciences (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: licensing
Action mechanism: genome editing product. Sangamo's zinc finger nucleases-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy approaches.
Disease: hemoglobinopathies
Details: * On November 30, 2016, Sangamo BioSciences announced that its collaborative zinc finger nuclease (ZFN)-mediated genome editing program for hemoglobinopathies will transfer to Bioverativ. In 2014, Sangamo and Biogen entered into an exclusive worldwide research, development and commercialization collaboration and license agreement under which both companies agreed to develop and commercialize therapeutic genome editing products for the potential treatment of two inherited blood disorders, sickle cell disease and beta-thalassemia. Bioverativ, the planned spin-off of Biogen's hemophilia business, is on track to launch as an independent, publicly-traded biotechnology company in early 2017.
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