Demetrios Braddock, MD, PhD, professor of pathology and principal investigator of the Braddock Laboratory, was recently honored by the patient community for leading the development of a new therapy for rare genetic neonatal calcification disorders.
He received the 2026 Hope Award from Brighter Hope Foundation (formerly known as GACI Global) during the organization’s international patient conference held in Orlando, Fla., on Valentine’s Day. Braddock traveled to attend the conference, where families, patients, and researchers gathered to share updates, build connections, and recognize progress in the field.
The Hope Award honors individuals who have made a meaningful impact on the lives of patients and families affected by rare genetic calcification disorders known as ENPP1 deficiency and ABCC6 deficiency. It reflects the community’s sentiment of “giving hope where there was none.”
Braddock invented BMN-401 (previously INZ-701), a therapy designed to address the underlying biology of rare calcification disorders such as ENPP1 deficiency and ABCC6 deficiency. These disorders can cause Generalized Arterial Calcification of Infancy (GACI), a rare and often life-threatening condition characterized by abnormal calcium buildup in the arteries. This begins as early as the second trimester of fetal development, resulting in death in approximately 50% of affected infants in the first 6 months of life. Survivors go on to develop a range of complications, including joint and bone pain, reduced mobility, rickets, hearing loss, fatigue, and more. Historically, treatment options have been limited, and outcomes for affected infants have been poor.
Braddock was the scientific founder of Inozyme Pharma. The company’s lead program came from his lab’s invention and published preclinical validation of the enzyme therapy for GACI, which was licensed to Inozyme and called INZ-701. After BioMarin Pharmaceutical acquired Inozyme Pharma, the program was renamed BMN-401.
The therapy is currently in late-stage clinical development, with Phase 3 studies recently completed. Some patients within the Brighter Hope Foundation community have participated in clinical trials or received treatment through expanded access programs, representing an important step forward for families affected by these conditions.
The foundation recognized Braddock not only for scientific innovation, but also for his persistence in advancing the work despite early challenges, including limited funding and skepticism due to the extreme rarity of these conditions.
While expressing gratitude for the recognition, Braddock emphasized that the most meaningful part of the experience was connecting directly with families. Meeting children who may benefit from therapy, he notes, was deeply moving, and underscored the importance of continuing his work.