Nick Goeden

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biotechnology aav engineering gene therapy biopharmaceuticals protein engineering analytical development process development aav immunology aav capsids cns targeting disease modification clinical development intravenous administration engineered capsids preclinical studies manufacturing processes neurodegenerative diseases therapeutic index gene delivery capsid engineering patient-centered approach strategic collaborations orphan drug designation disease modifying therapies stxbp1 encephalopathy parkinson's disease friedreich's ataxia preclinical proof of concept cargo optimization high-throughput screening neuron transduction disease-targeted therapies automated screening translational biology genetic medicine clinical trials biotechnology solutions contract manufacturing quality control patient advocacy central nervous system therapeutic expression safety profiles biopharmaceutical innovation unique capsid design elevated neuronal expression diminished liver targeting neurodevelopmental disorders non-invasive gene therapies community engagement regulatory expertise biotech partnerships innovative treatment strategies multidisciplinary approach long-term therapy efficacy targeted therapies cns disorders adeno-associated virus gene replacement gene editing gene silencing disease-modifying treatments rare diseases stxbp1 developmental encephalopathy proprietary platform safety profile manufacturing capabilities biopharmaceutical partnerships patient-centric approach innovative therapies comprehensive development therapeutic potential non-invasive treatments gene therapy pipeline collaboration with abbvie crispr therapeutics biotechnology industry precision medicine health care health wellness & fitness hospital & health care mechanical or industrial engineering