With deep expertise in RNA biology and translational gene therapy, I support biotech and research teams developing advanced therapies for rare genetic and neuromuscular diseases. My work focuses on bridging early discovery with clinical relevance through biomarker innovation and scalable delivery strategies. At Nationwide Children’s Hospital, I serve as a principal investigator in the Jerry R. Mendell Center for Gene Therapy and as an assistant professor at The Ohio State University College of Medicine. My research spans extracellular vesicle (EV)-based biomarker discovery and viral/non-viral gene therapy platforms, including RNA- and CRISPR-based approaches for muscular dystrophies, progeria, and related disorders. I also contribute to the field through leadership roles in ASGCT committees and translational programs aimed at improving tissue specificity, efficacy, and real-world readiness of gene therapies.
GENE THERAPY TRANSLATIONAL RESEARCH LEAD
Nizar Saad, BS, MS, Ph.D.
Expert advisor in RNA biology, gene therapy, and translational rare disease research, with strong experience in extracellular vesicle biomarker discovery and development of viral and non-viral therapeutic platforms for neuromuscular disorders.
Assistant Professor at The Ohio State University, Principal Investigator at the Center for Gene Therapy at Nationwide Children’s Hospital
LinkedIn profileAreas of expertise
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RNA biology & gene therapy
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Extracellular vesicle biomarkers
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Rare disease translational research
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Viral & non-viral delivery
