Micah Mackison

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biotechnology research rna therapies antisense oligonucleotides neurodegenerative diseases huntington's disease spinocerebellar ataxia polyglutamine disorders gene targeting molecular neuroscience clinical development allele-preferential targeting cag repeat expansion therapeutic development drug discovery biotechnology neuroscience research genetic medicine csf biomarkers huntingtin-lowering therapy precision medicine patient outcomes clinical trials rare genetic disorders mhtt reduction nucleoside chemistry oligonucleotide synthesis therapeutic innovation drug pipeline first-in-class therapies safety & tolerability chd modulating therapies biopharmaceuticals clinical-stage therapies preclinical studies patient-centric approach investigational medicines neuroprotective strategies molecular therapeutics neurological pathology neurodegeneration healthcare investment biotech investment research collaborations orphan drug designation multidisciplinary teams biotech ecosystem therapeutic strategies clinical endpoints health care health wellness & fitness hospital & health care