Gerald Cox, MD, PhD

Gerald F. Cox, MD, PhD
Chief Medical Officer
Owner, Gerald Cox Rare Care Consulting, LLC
Staff Physician in Genetics, Part-time, Boston Children’s Hospital
Instructor in Pediatrics, Harvard Medical School

Dr. Gerry Cox is a 20-plus year veteran of the biotechnology industry, and he has been a practicing clinical geneticist at Boston Children’s Hospital for over 25 years. Dr. Cox’s introduction to drug development began at Genzyme in 2000, where as the company’s first clinical geneticist, he rose to Vice President of Clinical Development for Rare Diseases. During his 16 years at Genzyme and later at Sanofi Genzyme, he led the clinical development programs of several enzyme replacement and substrate reduction therapies for lysosomal storage disorders. He played a key role in the approvals of treatments for MPS I (Aldurazyme), MPS II (Elaprase, Japan), Gaucher disease type 1
(Cerdelga), and Gaucher disease type 3 (Cerezyme, China). He also led the early clinical trials of treatments for infantile Pompe disease (Myozyme) and Niemann-Pick disease type B (Xenpozyme), which were subsequently approved. From 2016-2018, Dr. Cox was Chief Medical Officer at Editas Medicine, a CRISPR-based genome editing company. He led the first IND for the in vivo delivery of a CRISPR-based medicine, EDIT-101, to treat Leber congenital amaurosis type 10, the
most common cause of genetic blindness in children.
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Dr. Cox is a board-certified medical geneticist (clinical, biochemical, and molecular genetics) who continues to teach fellows and see patients on a part-time basis at Boston Children’s Hospital, where he was previously on the full-time staff and completed his pediatrics and genetics training. 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 in 1989, and a BA in biology magna cum laude from Harvard College in 1980. He has authored over 90 peer-reviewed publications and written several book chapters and reviews. In 2019, Dr. Cox joined the board of directors of the National Tay-Sachs and Allied Diseases patient advocacy organization, and in 2023, he joined the Scientific and Medical Advisory Boards of NORD, the Barth Syndrome Foundation, and CTNNB1 Connect and Cure.

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