EvGenTx Founder 

Dr. Brandon M. Henry is a physician–scientist with over a decade of translational and clinical research experience spanning cell, gene, and RNA therapy, as well as immunology, biomarkers, and evidence-based medicine. His work bridges academic and industry domains, with a focus on advancing clinical development strategies for rare and ultra-rare genetic diseases, where innovative therapeutic gene therapy approaches can transform previously untreatable conditions. 

With recognized expertise in adaptive and biomarker-driven trial design, Dr. Henry has led programs integrating enrichment strategies, biomarker-based endpoints, and companion diagnostics to accelerate the translation of novel genetic therapies from bench to approval. He brings extensive experience navigating the global regulatory landscape for biologics and advanced therapeutics and in designing and operationalizing complex, high-stakes clinical trials spanning early through late phases and across diverse patient populations—from neonates to adults.

Dr. Henry’s research and development experience includes AAV-based gene therapies, gene editing, mesenchymal stem cells, CAR-T and other adoptive cell therapies, oligonucleotide therapeutics, and RNA and DNA vaccine platforms, spanning preclinical translation through early- to late-phase clinical development.

He has authored more than 400 peer-reviewed publications, delivered invited lectures around the world, and received numerous honors for his contributions to translational science. He is widely recognized for his work in gene therapy safety, biomarker-driven trial design, and clinical evidence generation, and his research has informed the design and monitoring of multiple first-in-human AAV and gene-editing trials across neurodevelopmental, neuromuscular, and neurodegenerative disorders, as well as cardiovascular, oncologic, and metabolic diseases, including lysosomal storage disorders and other inborn errors of metabolism.

He is an elected IFAPP Senior Fellow—a distinction awarded for scientific leadership in pharmaceutical medicine—and has been recognized among the World’s Top 2% Scientists (Stanford / Elsevier, 2021–2025) and Clarivate Highly Cited Researchers (2024).

An active contributor to the global scientific community, Dr. Henry serves on several editorial boards and participates in international working groups shaping the future of pharmaceutical and translational medicine, including appointments to the External Affairs and Pharmacovigilance Working Groups of the International Federation of Associations of Pharmaceutical Physicians and Pharmaceutical Medicine. 

During the COVID-19 pandemic, he served as Chairman of International Federation of Clinical Chemistry and Laboratory Medicine Working Group on SARS-CoV-2 Variants and led several global efforts to evaluate immunogenicity of mRNA vaccines using novel assays.

Dr. Henry’s professional mission is to advance the science and safety of genetic therapeutics, applying rigorous evidence synthesis and translational insight to ensure that innovative treatments achieve meaningful and responsible patient impact.