Ocular gene therapy for neovascular AMD: a new era? – Authors' reply

To mitigate risk of vision loss from AAV2-sFLT01 or withholding of standard care, only patients with subretinal fibrosis and hence limited visual potential were eligible for cohorts 1-4; however, presence of intraretinal or subretinal fluid was desired to allow assessment of anti-permeability activi...

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Bibliographic Details
Published in:The Lancet (British edition) 2017-11, Vol.390 (10108), p.2140-2140
Main Authors: Campochiaro, Peter A, Heier, Jeffrey S, Kherani, Saleema, Le-Halpere, Annaig, Scaria, Abraham
Format: Article
Language:English
Subjects:
Choroidal Neovascularization
Fees & charges
Gene therapy
Genetic Therapy
Humans
Macular Degeneration
Patients
Permeability
Pharmaceuticals
Vascular endothelial growth factor
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Summary:To mitigate risk of vision loss from AAV2-sFLT01 or withholding of standard care, only patients with subretinal fibrosis and hence limited visual potential were eligible for cohorts 1-4; however, presence of intraretinal or subretinal fluid was desired to allow assessment of anti-permeability activity. Some patients with advanced disease might be incapable of showing benefit, and therefore once three patients received 2 x 1010 vector genomes of AAV2-sFLT01 without adverse effects, we changed the eligibility criteria to allow enrolment of patients with less severe disease and greater visual potential. SK declares no competing interests. 1 JS Heier, S Kherani, S Desai, Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial, Lancet, Vol. 390, 2017, 50-61
ISSN:0140-6736
1474-547X
DOI:10.1016/S0140-6736(17)32424-8