Dr. Brandon M. Henry, M.D., is a physician–scientist specializing in the clinical development of gene replacement, gene editing, and RNA therapies for rare and ultra-rare diseases. His work spans academic, biotech, and nonprofit settings, with a focus on designing and executing integrated regulatory and clinical development strategies for neurodevelopmental, neurodegenerative, neurometabolic, and neuromuscular disorders, where these approaches have the potential to fundamentally alter disease trajectory.

Dr. Henry is the founder of Aurelix Bio, where he provides embedded clinical development and regulatory leadership across multiple drug development programs, guiding assets from early translational stages through IND submission and into clinical development with registration intent. He serves as the Chief Medical Officer of the FOXG1 Research Foundation, leading clinical strategy, trial design, and regulatory execution for a clinical-stage CNS-directed AAV gene therapy program, and holds key leadership roles across several additional nonprofit and biotech organizations advancing gene-directed therapies for rare diseases.

He brings deep expertise in AAV vector biology, gene editing, RNA therapeutics, CNS-directed delivery, immune risk mitigation, and long-term safety strategy, with extensive experience designing complex pediatric trials. His work emphasizes integrated clinical, nonclinical, regulatory, and CMC alignment, novel endpoint development, and innovative trial designs, including external comparator frameworks, to enable efficient and credible paths to registration in rare and ultra-rare diseases.

Previously, Dr. Henry served as Medical Director of the Cell and Gene Therapy Center of Excellence at IQVIA, where he led medical and regulatory strategy for global gene-directed therapy programs across multiple indications. He has authored more than 400 peer-reviewed publications and has been recognized among the World’s Top 2% Scientists (Stanford/Elsevier) and as a Clarivate Highly Cited Researcher, reflecting sustained impact in translational and clinical science.

His research focuses on translating complex gene-directed therapy science into rigorous, patient-centered clinical programs that enable the responsible and efficient advancement of transformative therapies for patients with severe genetic diseases.