In the first session of this year’s Pathology Innovation & Entrepreneurship series, Yves Sabbagh, PhD, took an audience of Yale postdocs, research scientists, faculty, and other researchers inside the realities of developing biotech therapies for rare bone and mineral disorders. Using case studies from Genzyme, Inozyme, and now BioMarin, he showed how scientific insights become drug programs, when to make hard go/no-go calls, and how a career can progress from bench scientist to R&D leader in industry. Organized by Demetrios Braddock, MD, PhD, a faculty member in Pathology and founder of Inozyme, which was acquired by BioMarin in 2025, the series is co-hosted by the Yale Department of Pathology and Yale Ventures. 

“No dragging failed projects forever—the worst thing you can do is waste people’s time and money. You’ll see more success by making a firm decision to cancel early and move forward.”—Yves Sabbagh, PhD 

Session Highlights

Career decisions in real time

Sabbagh traced his path from postdoc work on phosphate metabolism and rare bone diseases into industry roles at Genzyme, a startup (Inozyme), and BioMarin, emphasizing how conferences, networking, and staying in touch with collaborators opened key doors.

Target validation & project triage

He shared his first major industry project on phosphate transporters, illustrating how robust target validation, animal models, and early data guide decisions to advance or terminate programs before they consume excessive resources.

Learning from clinical trials

Through examples in conditions such as achondroplasia, X-linked hypophosphatemia, and osteogenesis imperfecta, he highlighted the difficulty of translating animal data to human outcomes, the constraints of working with limited rare disease populations, and the importance of designing trials that generate clear, interpretable data.

Building and expanding a pipeline

At Inozyme, Sabbagh helped build a rare disease pipeline, including a drug for ENPP1 deficiency that increased pyrophosphate levels, reduced calcification, and improved functional outcomes like six-minute walk tests. He discussed expanding indications to ABCC6 deficiency, calciphylaxis, and craniofacial dysplasia by thinking broadly about disease mechanisms.

Strategic leadership in biotech

Reflecting on InDesign’s acquisition by BioMarin, he underscored how collaborations, publications, and focused pipelines help position a company for growth. He also spoke about leading bone-targeted drug programs in his current role and the mindset shift required to lead across multiple programs and teams.

About the Speaker

Yves Sabbagh, PhD, began his career studying phosphate metabolism and rare bone diseases, creating one of the first mutation databases for X-linked hypophosphatemia (XLH) during his PhD. A postdoctoral fellowship at Harvard expanded his in vivo work on vitamin D receptor knockout models and the impact of serum phosphate on bone growth. He joined Genzyme in 2005 and rose to head of the rare bone disease unit, leading programs in phosphate regulation and TGF beta–targeted therapies through mergers and acquisitions, including Sanofi’s acquisition of Genzyme. In 2020, he became Chief Scientific Officer and senior VP at Inozyme, guiding a rare disease pipeline and directing multiple clinical trials in ENPP1 and ABCC6 deficiencies and CKD before the company’s acquisition by BioMarin in July of 2025, where he continues to develop therapies in the bone mineralization space and mentor the next generation of scientists.