Improving Rare Disease Awareness: Bruce Dezube, MD

Video

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.

“Rare disease brings together government agencies, including both the funding and the regulatory agencies, pharmaceutical industries, and patient advocates. And, their priorities can sometimes differ... These different groups are involved with every aspect [of therapy development] and every aspect is working, but every aspect could be working even better.”

Monday, February 28, marked international Rare Disease Day, which recognizes over 7000 rare conditions that affect over 300 million people worldwide. The day brought together researchers, patients, advocates, and policy makers in conversations on how to improve awareness and management of rare diseases.

Severe combined immunodeficiency (SCID), glioblastoma, and chronic lymphocytic leukemia (CLL) are 3 rare diseases that companies such as Mustang Bio are developing therapies for. These programs, lentiviral gene therapies in SCID and glioblastoma and chimeric antigen receptor (CAR) T-cell therapies in CLL, are in phase 1 studies with planned phase 2 parts.

CGTLive spoke with Bruce Dezube, MD, senior vice president and head, clinical development, Mustang Bio, to learn more about the importance of Rare Disease Day as well as the role of collaboration and advocacy to improve rare disease awareness and therapy development.

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.