Nonviral Gene Transfer to the Suprachoroidal Space

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Unmet Need:
Gene transfer with adenoassociated viral (AAV) or lentiviral vectors can provide long-term expression in cells that have appropriate receptors for transduction. However, viral vectors induce an immune response making repeated administration impossible and also have limitations to cargo capacity. An alternative is nonviral gene transfer with biodegradable particles. However, transfection efficiency is generally substantially less with nonviral gene transfer compared with that of viral vectors.
Technology Overview:
Johns Hopkins researchers have overcome this problem by injecting nanoparticle and microparticle containing expression constructs into the suprachoroidal space. This is a potential space that is expanded by the injection keeping cells and particles in close proximity. This plus the nature of the cells in the suprachoroid results in good transfection and sustained expression after suprachoroidal injection of the particles. In addition, suprachoroidal injections are relatively noninvasive and can be done in an outpatient clinic setting making repeated injections feasible.
The key aspect of the invention is that targeting the suprachoroidal space makes nonviral gene transfer with nanoparticles and microparticles feasible for ocular gene transfer providing durable transgene expression that can be augmented by repeated injections. This provides a novel approach for sustained delivery of proteins and peptides to the retina and choroid. Elements of the invention also cover particular biomaterial and particle compositions and formulations useful for ocular delivery.
Stage of Development:
The inventors demonstrate that it is possible to transfect photoreceptors, Muller cells, RPE, and choroid by suprachoroidal injection of an expression construct packaged in polymeric nanoparticles.

Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
NONVIRAL GENE TRANSFER TO THE SUPRACHOROIDAL SPACE PCT: Patent Cooperation Treaty United States 16/753,245   4/2/2020     Pending
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Christine Joseph
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