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Drug Names(s): adeno-associated viral vector serotype 2, AAV2.REP1
Description: AAV.REP1 is an adeno-associated viral (AAV) vector containing the Rab escort protein-1 (REP1) gene. Patients with choroideremia have defects in REP1 that cause progressive retinal damage and blindness.
Isis Innovation and Nightstar
In January 2014, Syncona announced a 12 million investment in NightstaRx Ltd a spin-out from the University of Oxford and its research commercialisation company Isis Innovation. The companys first programme is a gene therapy for an inherited form of progressive blindness called choroideremia AAV.REP1, was developed by a team of researchers, led by Professor Robert MacLaren of the University of Oxford, and is currently being studied in a 12 patient Phase I clinical trial supported by the Wellcome Trust and Department of Health. Nightstar has an exclusive licence from Isis Innovation to the intellectual property which underpins this program.
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