Dr. Gerry Cox is Vice President of Rare Diseases Clinical Development at Sanofi Genzyme, Cambridge, MA. Since joining Genzyme in 2000, Dr. Cox has overseen the clinical development programs for several lysosomal storage disorders and was the medical lead for the regulatory approvals of two enzyme replacement therapies (Aldurazyme for MPS I in 2003, and Elaprase for MPS II in Japan in 2006) and one substrate replacement therapy (Cerdelga for Gaucher disease type 1 in 2014). His group also is developing olipudase enzyme replacement therapy for Niemann-Pick B disease (Phase 3) and a novel CNS-penetrant glucosylceramide synthase inhibitor for Gaucher disease type 3 and several other indications (Phase 2). He holds two patents related to the treatment of Niemann-Pick B disease. Dr. Cox is a practicing physician who is board-certified in clinical, biochemical, and molecular genetics, and formerly in pediatrics. Prior to Sanofi Genzyme, he was a full-time staff geneticist at Boston Children’s Hospital, where he continues to see patients part-time and train fellows in the genetics clinic. He is an instructor in pediatrics at Harvard Medical School and was a founding member of the scientific advisory boards for the Barth Syndrome Foundation and the Pediatric Cardiomyopathy Registry.
Dr. Cox graduated magna cum laude from Harvard College in 1980 with a B.A. in biology. He completed the Medical Scientist Training Program at the University of California at San Diego, where he received his M.D. and a Ph.D. in biology in 1989. His PhD thesis under Dr. S.J. Singer characterized the immunocytochemical localization of two novel cytoskeletal proteins (zeugmatin and enactin) associated with adherens junctions and sarcomeres. He then went to Boston Children’s Hospital, where he completed an internship and residency in pediatrics, and clinical and research fellowships in genetics. As a Howard Hughes post-doctoral fellow with Dr. Louis Kunkel, Dr. Cox researched dystrophin-related and associated proteins and linked the COL9A3 gene to autosomal dominant multiple epiphyseal dysplasia with mild myopathy.
Dr. Cox has authored more than 70 peer-reviewed publications, reviews, and book chapters. His clinical interests include inborn errors of metabolism and genetic causes of cardiomyopathy and eye diseases.