Gerald F. Cox

Dr. Cox is the founder of Gerald Cox Rare Care Consulting, LLC, where since 2018 he has been providing expert advice to small companies in all phases of clinical development for investigational rare disease drugs. From 2016-2018, Dr. Cox was the Chief Medical Officer of Editas Medicine, where he led the clinical development of CRISPR-based genome editing medicines to treat human diseases, including the first approved IND for a CRISPR-based medicine to be delivered in vivo that is designed to treat a genetic form of blindness called Leber congenital amaurosis type 10. Prior to Editas Medicine, Dr. Cox held increasingly senior roles at Genzyme (now Sanofi Genzyme) for over 15 years, advancing to Vice President of Rare Disease Clinical Development. While at Genzyme, he played an instrumental role in the global development and approval of treatments for several lysosomal storage disorders, including the enzyme replacement therapies Aldurazyme® (laronidase) for Mucopolysaccharidosis type I in 2003, Elaprase® (idursulfase) for Mucopolysaccharidosis type II in Japan and the Asia Pacific region in 2007, and Cerezyme® (imiglucerase) for a label expansion in Gaucher disease type 3 in Australia and China in 2016, as well as the substrate reduction therapy Cerdelga® (eliglustat) for Gaucher disease type 1 in 2014. He also led the early clinical development of the enzyme replacement therapies Myozyme® (alglucosidase alfa) for infantile Pompe disease, which was approved in 2006, and olipudase alfa for Niemann-Pick disease type B, which recently completed a successful Phase 3 study. Dr. Cox has been affiliated with Boston Children’s Hospital during his entire career, where he is a Part-time Staff Physician in Genetics. He is also an Instructor in Pediatrics at Harvard Medical School.

Dr. Cox received his MD and PhD from the University of California at San Diego and his B.A. from Harvard College. He completed an internship and residency in pediatrics followed by clinical and post-doctoral research fellowships in genetics at Boston Children’s Hospital and was Director of the Medical Genomics Mapping Facility. Dr. Cox is board-certified by the American College of Medical Genetics and Genomics in Clinical, Biochemical, and Molecular Genetics, and he was board-certified by the American Academy of Pediatrics in the past. He serves on the Board of Directors for the National Tay-Sachs and Allied Diseases organization.