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ZFP - Beta-Thalassemia
Drug Names(s): ZFP - Beta-Thalassemia, SB-BCLmR-HSPC
ZFP Therapeutic for beta-thalassemia uses Sangamo's zinc finger nuclease (ZFN) gene-editing technology to modify a patient's own hematopoietic stem cells (HSCs), thereby eliminating the need for a matched donor.
Normally in infancy, fetal hemoglobin fully protects beta-thalassemia patients from developing disease symptoms. Later in childhood however, production of fetal hemoglobin ceases and is replaced by synthesis of adult-type beta-globin chains that are defective in beta-thalassemia patients. ZFP Therapeutic enables a patient's cells to permanently produce therapeutic fetal hemoglobin to achieve normal levels of hemoglobin and RBCs. The company has not disclosed the precise genetic target.
The process involves mobilization of the patient's cells into the blood stream prior to collection.
Biogen and Sangamo
In January 2014, Biogen Idec and Sangamo BioSciences announced an exclusive worldwide collaboration and license agreement focused on the development of therapeutics for hemoglobinopathies, inherited conditions that result from the abnormal structure or underproduction of hemoglobin. The agreement will enable Biogen Idec to further enhance its expertise in non-malignant hematology by leveraging Sangamo's proprietary genome-editing technology platform to develop treatments targeting sickle cell disease (SCD) and beta-thalassemia.
Under the terms of the agreement, Sangamo is responsible for all research and development activities through the first clinical proof of concept trial in beta-thalassemia, and both companies will perform activities to enable submission of an Investigational New Drug (IND) application for SCD. Biogen Idec will be responsible for subsequent worldwide clinical development and commercialization of products arising from the...See full deal structure in Biomedtracker
Partners: Sangamo Biosciences, Inc.
ZFP - Beta-Thalassemia News
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