We are delivering Calcium/calmodulin-dependent protein kinase II (CaMKII) via singleAAV intravitreal injection to protect retinal ganglion cells (RGC). Our animal model showed that CaMKII plays a crucial role in RGC survival and function. CaMKII is involved in the regulation of axonal growth and guidance, synaptic plasticity, and cell survival. It is also implicated in the pathogenesis of various retinal diseases, including glaucoma and retinal ischemia.
We are inhibiting the surface exposed functional domain of vitronectin by delivering an antibody using AAV. This single AAV via IVT injection will also contain RNAi to silence the VTN expression inRPE. In AMD patients, VTN levels were elevated and it plays an essential role in the drusen formation. VTN interacts with several other molecules that are involved in AMD pathogenesis, including complement factor H (CFH) and complement factor I (CFI).
NRO-1 is a topically delivered, nerve regenerative therapeutic that accelerates nerve regeneration and recovery of functional nerves. It is a GDNF releasing small molecule that ameliorates corneal epithelial layer and it is positioned to initiate P2a trial for neurotrophic keratitis patients.
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