Ram Palanki

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biotechnology biopharmaceuticals gene therapy biotechnology: biological products (no diagnostic substances) health care biotechnology research aav gene therapy nav technology platform curative potential clinical-stage biotechnology gene delivery aav therapeutics retinal diseases duchenne muscular dystrophy mps ii rgx-314 rgx-202 rgx-121 patient outcomes one-time treatments long-lasting effects gene editing genetic disorders nav vectors drug development manufacturing regulatory affairs clinical programs research & development patient advocacy expanded access rare diseases metabolic disorders neurodegenerative diseases therapeutic pipeline innovative therapies healthcare solutions gene transfer clinical trials healthcare innovation regenerative medicine commercialization biotechnology firms medical research life sciences patient-centric care translational medicine pioneering science biotech collaborations corporate partnerships therapeutic innovations transformative medicine high-impact research adeno-associated virus aav vectors metabolic diseases innovative therapeutics healthcare professionals rare genetic diseases patient-focused scientific excellence collaboration mucopolysaccharidosis wet age-related macular degeneration diabetic retinopathy therapeutic proteins biotechnology industry partnerships patient demographics healthcare market genetic medicine therapeutic candidates fda approval orphan drug designation rare pediatric disease designation fast track designation gene therapy innovation compassionate care integrity in research market competition biotechnology sector health wellness & fitness mechanical or industrial engineering hospital & health care