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TECHNOLOGY
Targeting lncRNA for retinal disease
i-RNA is developing siRNA therapeutics targeting long non-coding RNAs involved in retinal disease, with a lead program in diabetic retinopathy designed for non-invasive ocular delivery.
OUR APPROACH
Target.
Silence.
Deliver.
Our approach combines lncRNA targeting, RNA interference and ocular delivery to address multiple pathways involved in diabetic retinopathy.
Target
Long non-coding RNAs
Our lead program targets HOTAIR, a long non-coding RNA upregulated in the diabetic retina that regulates multiple disease-associated factors, including VEGF.
Silence
RNA Interference
We use small interfering RNA (siRNA) to silence HOTAIR and modulate multiple disease-associated pathways involved in diabetic retinopathy.
Deliver
Ocular Delivery
Our lead program uses GalNAc-modified siRNA for ocular delivery, evaluated through both topical eye-drop and intravitreal administration
TARGETING HOTAIR
An upstream RNA target in diabetic retinopathy
HOTAIR (HOX transcript antisense intergenic RNA) is a long non-coding RNA involved in the regulation of gene expression. Our research has shown that HOTAIR is upregulated in the diabetic retina and regulates disease-driving pathways, including angiogenesis, inflammation and oxidative stress, and key factors such as VEGF.
By targeting HOTAIR upstream, our approach is designed to modulate multiple disease-associated pathways simultaneously.
Conceptual illustration of i-RNA’s therapeutic approach.
FLEXIBLE OCULAR DELIVERY
Two routes for ocular administration
i-RNA-DR2 is being developed as an RNA therapeutic for diabetic retinopathy, with the potential for both topical and intravitreal administration.
Intravitreal Injection
A direct route to the retina. HOTAIR-targeting siRNA has demonstrated target silencing and biological activity following intravitreal administration in preclinical models.
Eye Drop
Lead Approach
A non-invasive route to the retina. Topically administered GalNAc-modified siHOTAIR has demonstrated retinal localization, target silencing and biological activity in preclinical models.
PUBLICATIONS
Recent Publications
Our research has been published in leading scientific journals, demonstrating the potential of RNA-based diagnostics and treatment for retinal disease.
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