Angela Arafelis

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gene therapy adenoassociated virus biotechnology rare diseases clinical lab clinical trials science biotechnology: biological products (no diagnostic substances) health care biotechnology research genetic medicines ophthalmology pulmonary diseases cardiology diseases customized vectors directed evolution aav vectors wet amd diabetic macular edema cystic fibrosis intravitreal delivery aerosol delivery intravenous delivery therapeutic payloads r100 vector a101 vector c102 vector transformative therapies patient-focused genetic medicine pipeline curative intent eye diseases lung diseases heart diseases vascular endothelial growth factor vegf treatment retinal diseases neovascular retinopathies clinical development product candidates safety & tolerability clinical proof of concept disease modification biopharmaceuticals patient advocacy unmet medical needs therapeutic vector evolution disease-specific payloads phase 1/2 clinical trials fda fast track designation biotech partnerships quality of life improvement efficacy results biotech innovation interdisciplinary collaborations healthcare solutions innovation in medicine gene delivery adeno-associated virus (aav) pulmonology cardiology wet age-related macular degeneration fabry disease x-linked retinitis pigmentosa choroideremia clinical-stage biotechnology patient collaboration innovative treatments healthcare professionals genetic disorders biotechnology partnerships patient outcomes precision medicine therapeutic areas durable therapies disease-targeted treatments advanced gene therapies healthcare innovation health wellness & fitness hospital & health care medical