Liz (Stone) Smith, PhD
Senior Scientist @ Sarepta Therapeutics
About
I am a PhD-trained neuroscientist and strategic R&D leader with 5+ years of biopharmaceutical experience directing preclinical development and IND-enabling strategy for gene therapy and biologics programs in rare disease. My work sits at the intersection of rigorous science, clear strategy, and operational execution—with a consistent focus on advancing early-stage programs toward the clinic. At Sarepta Therapeutics, I served as a nonclinical lead and people manager, owning preclinical strategy across multi-species GLP and non-GLP toxicology, pharmacology, PK/PD, biodistribution, and biomarker development. I have led complex portfolios spanning multiple species and transgenic models, managed CROs and external collaborators, and delivered regulatory-ready data packages aligned to program and governance milestones. What differentiates my approach is an ability to translate complexity into clarity—without oversimplifying or gatekeeping the science. I partner closely with Clinical, Regulatory, CMC, Program, and Research teams to ensure nonclinical strategy is integrated, data-driven, and clearly understood across functions. I have presented strategy and data to scientific advisory boards, senior governance, and cross-functional leadership, and I am comfortable navigating both detailed execution and high-level decision making. I bring deep experience in gene therapy nonclinical development, including novel biomarker and imaging workflows, advanced biodistribution strategies, and first-in-class program design. Equally important, I am recognized as a calm, collaborative leader who builds high-performing teams, mentors scientists, and creates environments of ownership, accountability, and trust. I have directly managed and developed scientists and supervisors, led lab and infrastructure expansions, and driven process improvements that strengthened quality, communication, and operational efficiency. Colleagues know me as someone who is: - Grounded in data, but strategic in perspective - Clear, direct, and thoughtful in communication - Collaborative across functions and modalities - Willing to lead from the front when execution matters I am particularly interested in Associate Director–level roles in Preclinical Development, Nonclinical Strategy, or Early Development Leadership, where I can help shape integrated development plans, guide teams through complexity, and advance high-quality science into the clinic. 📍 Columbus, OH | Open to hybrid or remote roles 💬 Always happy to connect with others working in early development, gene therapy, and rare disease
United States
Westerville
Research
DNA Extraction, Drug Development, Gel Electrophoresis, Molecular Cloning, Cross-functional Team Leadership, Cross-functional Collaborations, SOP Development, Publications, Report Writing, Regulatory Strategy, Continuous Process Improvement, Fluorescence Microscopy, Good Manufacturing Practice (GMP), Good Laboratory Practice (GLP), Tissue Processing, Droplet Digital PCR (ddPCR), Western Blotting, ELISA, Microdissection, Pharmacology
Experience

Senior Scientist
Columbus, Ohio, United States
Served as the preclinical research lead for developing a novel gene therapy for peripheral neuropathies; developed and designed a preclinical strategy and all requisite animal studies, experiments, and biomarkers to fulfill the planned strategy; served as a subject matter expert on gene therapy preclinical development, equipment onboarding, and technique development; served as a study director, study monitor, and principal investigator for internal and external preclinical animal studies required for advancing drug development; fostered collaboration within and without our department by hosting and proposing meetings and representing the department in projects aimed to improve processes; and led a diverse team of scientists and research associates in a wide range of preclinical activities including biomarker development, advanced imaging acquisition and analysis, and biodistribution assessments

Scientist II
Columbus, Ohio, United States
Served as a subject matter expert on gene therapy preclinical development, technique development, and onboarding; served as the preclinical research lead for a novel gene therapy designed for peripheral neuropathies; served as a study director for preclinical animal studies needed for advancing our gene therapies; conducted any applicable and necessary laboratory activities to support ongoing research; authored study protocols, study reports, manuscripts, standard operating procedures, and any other required documents; drove forward collaboration and process improvement efforts needed to facilitate the best and most efficient working environment for my team; and managed a team of research associates

Scientist I
Dublin, Ohio, United States
Served as a subject matter expert on technique development and onboarding; conducted applicable and necessary laboratory activities to support ongoing research with focus on necropsy and imaging, served as a study director for preclinical animal studies needed for advancing our gene therapies for myopathies, and authored study protocols, study reports, standard operating procedures, as well as any other required documentation

Science Writer
Charcot-Marie-Tooth Research Foundation
On a contract basis I wrote original pieces and performed some editing/fact checking for the website of the Charcot-Marie-Tooth Research Foundation (CMTRF) website. These pieces are aimed at a general audience and function to provide more information about the various forms of Charcot-Marie-Tooth disease and point out their differences and similarities.

Interpreter
Cape Cod National Seashore
Informed the public on current conservation efforts for specific shore-nesting birds. I was involved in some of the conservation efforts as well - this mostly consisted of patrolling the nesting sites and recording the numbers of nests, adult pairs, and chicks/eggs.
Education
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