Dr. Christian Mueller is a distinguished researcher whose expertise in gene therapy has propelled significant advances in the treatment of rare genetic disorders. He previously held a tenured position as Associate Professor in the Department of Pediatrics and the Horae Gene Therapy Center at the University of Massachusetts Medical School, where he was awarded tenure in 2018. His academic foundation includes a PhD in Genetics from the University of Florida College of Medicine followed by postdoctoral training as a Parker B Francis Fellow while completing his Master's in Clinical Investigation at UMass Medical School. This rigorous academic preparation enabled him to establish the Mueller Lab for Gene Therapy in 2011, creating a center dedicated to developing innovative therapeutic approaches for challenging genetic conditions.
Dr. Mueller pioneered critical advancements in adeno-associated virus (AAV) mediated gene therapy, being the first to describe regulatory T-cell responses to AAV capsids in patients and developing the use of AAV to deliver artificial miRNAs for targeted gene silencing. His laboratory's development of dual-function AAV vectors represents a paradigm shift in therapeutic design, enabling simultaneous augmentation of normal protein expression while silencing mutant proteins—a breakthrough approach particularly valuable for conditions like alpha-1 antitrypsin deficiency. This innovative work earned recognition in 2017 for holding one of the top 10 patents in Massachusetts. With over 75 peer-reviewed publications and 10 patent applications, his research has established foundational knowledge that is now guiding clinical development for multiple rare diseases including amyotrophic lateral sclerosis and Huntington's disease.
Beyond his laboratory achievements, Dr. Mueller has emerged as a respected thought leader in the gene therapy community, frequently invited to present at major scientific conferences worldwide. His insights on immune responses to viral vectors have provided critical understanding that informs the safety profiles of emerging gene therapies. As a dedicated mentor, he has trained numerous researchers who now contribute to the field across academia and industry. Following his tenure at UMass, he served as Head of Gene Therapy at Sanofi and currently leverages his expertise as Co-Founder and Scientific Advisor at Apic Bio, continuing to advance the translation of his dual-function vector platform into clinical applications for patients with previously untreatable genetic conditions.
