Jared Smith
Principal Scientist @REGENXBIO
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WORK HISTORY
Principal Scientist @REGENXBIO
Leading a team in biotech to develop AAV-based approaches to gene therapy for a variety of diseases (epilepsy, neurodevelopmental, neurodegenerative, and neuromuscular). • Conduct pre-clinical animal studies across a range of mammalian species (from rodent to primate) to generate proof of concept data to support clinical candidate IND submissions. • Collaborate on cross-functional platform development studies to design and validate new AAV technologies such as novel capsids and promoters, novel routes of administration, transgene expression kinetics/durability studies (e.g. Hollidge et al) and more • Leading key infrastructure developments including a cutting-edge microscopy core as well as designing, building, and directing a fully staffed animal research facility for pre-clinical studies • Key contributor to corporate partnership developing a clinical candidate for AAV gene therapy of temporal lobe epilepsy which was sold for >$40M (Corlieve sold to UniQure) • Member of early pipeline portfolio planning team for CNS, neuro-muscular, and ocular indications • Presented scientific findings at international conferences (ASGCT, ESGCT, etc.), invited talks, and publishing numerous manuscripts • Collaborated with academic and corporate partners on numerous technology development projects (e.g. novel promoter for targeted expression in subtypes of GABAergic neurons – Niibori et al; novel imaging modality for unbiased assessment of body-wide AAV expression - Endpoints News webinar) • Collaborated with clinical operations, patient advocacy and the pediatric ethics group at NYU/Mt. Sinai to write an ethics paper on the lived experience of patients and families in clinical trials • Edited two special issues of Frontiers in Neurology and Frontiers in Systems Neuroscience on gene therapy approaches to treating diseases of the basal ganglia • Served as a reviewer for numerous journals and grant funding agencies (including MJFF and others)
EDUCATION
Penn State College of Medicine
Doctor of Philosophy (Ph.D.), Neuroscience
Bucknell University
Bachelor of Science - BS, Chemistry
SKILLS
ABOUT JARED SMITH
5+ years in biotech leading research groups to develop AAV gene therapies in epilepsy…
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