TJ Cradick

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gene editing crispr gene therapy genome medicine biotechnology research crispr-cas12 crispr-cas13 gene replacement gene modification rna editing genomic medicine biotechnology biopharma neurology ophthalmology neuroscience clinical trials therapeutic programs rare diseases orphan drug designation genome editing high-throughput screening preclinical development manufacturing capabilities animal models gene delivery genetic disorders disease focus neovascular age-related macular degeneration rpe65 mutation mecp2 duplication syndrome duchenne muscular dystrophy huntington's disease amyotrophic lateral sclerosis biological drugs research & development patent application intellectual property biomedicine healthcare products precision medicine clinical development fda approval cro partnerships technology licensing patient care cell therapy therapeutic proteins gmp production innovative therapies health care health wellness & fitness hospital & health care