2020
DOI: 10.1089/jop.2019.0126
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Suprachoroidal Delivery of Viral and Nonviral Gene Therapy for Retinal Diseases

Abstract: Retinal gene therapy is a rapidly growing field with numerous clinical trials underway, and route of delivery is a critical contributor to its success. Subretinal administration, which involves pars plana vitrectomy in the operating room, offers targeted delivery to retinal-pigment epithelium cells and photoreceptors. Due to the immune-privileged nature of the subretinal space, the risk of an immune reaction against viral capsid antigens is minimized, an advantage of subretinal administration in patients with … Show more

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Cited by 45 publications
(55 citation statements)
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“…Microneedles are made to the length of the approximate thickness of the sclera, and are used with a custom injector specifically designed for suprachoroidal drug delivery [3][4][5][6][7]. While there are some challenges with this type of suprachoroidal injection procedure, including the lack of direct intraoperative visualization and the potential need for customized injectate formulations that are compatible with the device [26,32], to date, the procedure has been performed over 1,000 times in clinical trial patients with an acceptable safety profile [33]. Suprachoroidal injection using proprietary microneedlebased technology, the SCS Microinjector ® (Clearside Biomedical, Alpharetta, GA) consists of three key steps, unique from other ocular injections.…”
Section: Methods Of Accessing the Scsmentioning
confidence: 99%
“…Microneedles are made to the length of the approximate thickness of the sclera, and are used with a custom injector specifically designed for suprachoroidal drug delivery [3][4][5][6][7]. While there are some challenges with this type of suprachoroidal injection procedure, including the lack of direct intraoperative visualization and the potential need for customized injectate formulations that are compatible with the device [26,32], to date, the procedure has been performed over 1,000 times in clinical trial patients with an acceptable safety profile [33]. Suprachoroidal injection using proprietary microneedlebased technology, the SCS Microinjector ® (Clearside Biomedical, Alpharetta, GA) consists of three key steps, unique from other ocular injections.…”
Section: Methods Of Accessing the Scsmentioning
confidence: 99%
“…The standard for ocular gene therapy administration is subretinal injection, a highly-specialized surgical procedure, which is generally performed at selected Centers of Excellence, given the potential complications of pars plana vitrectomy associated procedures such as retinotomy [ 78 , 79 ]. Suprachoroidal gene delivery, an alternative route of administration, has demonstrated promise preclinically and is currently undergoing clinical evaluations [ 80 ]. Delivery of gene therapy via SC injection enables an in-office gene therapy administration and may facilitate increased patient access as well as reduced procedural risks compared to subretinal delivery.…”
Section: Viral and Nonviral Gene Therapiesmentioning
confidence: 99%
“…However, the suprachoroidal space is not anatomically immune privileged and the presence of choriocapillaris causes rapid clearance of the administered drug hindering effective transduction of the retina. 96 The Choice of AAV Vector AAV, a parvovirus, has a protein capsid surrounding a DNA genome (single stranded) approximately 4.8 kb in size. The genome contains genes responsible for replication, capsid, and assembly.…”
Section: Rpe65 (Lca 2 Locus)mentioning
confidence: 99%