Sangamo Therapeutics Inc. may be months away from demonstrating that its gene therapy for hemophilia A – SB-525, partnered with Pfizer Inc. – offers best-in-class sustainability of its therapeutic effect, which is crucial because competitor BioMarin Pharmaceutical Inc. is positioned to get to market first with a gene therapy for the disease.
The potential of showing “reliability” represents Sangamo’s best chance to overcome BioMarin’s expected first-to-market advantage, CEO Sandy Macrae said at the J.P. Morgan Healthcare Conference on 16 January. The exec has previously explained that an optimal gene therapy for hemophilia A – replacing regular treatment with Factor VIII – must be “safe, reliable and predictable
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