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Neuronal Barrier Modulation Modulating the Blood-Brain & inner Blood Retinal Barrier Basic overview Applications •Up to 98 % of low molecular weight drugs do not passively diffuse across the Blood-brain barrier (BBB) or inner Bloodretina barrier (BRB). This platform technology could potentially allow for the treatment of a wide variety of diseases that are to date untreatable; •The BBB and BRB contain tight-junctions (TJ‟s). Neurological degenerations such as Alzheimer disease, Parkinson disease, MS, motor neuron disease, and other degenerative conditions of the brain. Diabetic and hereditary retinopathies using anti‐neovascular and neuro‐protective drugs, Age-related macular degeneration (AMD). •Highly selective barrier formed. What Problem does it Solve & Advantages •Many FDA and EMEA‐approved low molecular weight drugs cannot be administered systemically due to the impermeable nature of the neuronal barriers, the BBB and iBRB. •Drug access is blocked by the tight architecture of the vascular endothelial cells of the blood vessels supplying the brain and retina. Brain malignancies, where access to the brain of chemotherapeutic agents is notoriously inefficient because of the inability of most of these drugs to cross the blood‐brain barrier. Traumatic brain injury (TBI), where a combination therapy of systemically administered siRNA targeting claudin‐5, together with mannitol, may radically improve survival by reducing brain edema. US DoD funded. (Telemedicine & Advanced Technology Research Center, Fort Detrick (TATRC). •Our technology targets one specific protein at the tight junction termed “claudin-5”. Allows small molecules to diffuse from the blood to the brain or retina, excluding larger potentially harmful substances. Technology and Patent Status •PCT application published on April 16th 2009. An International search report published in April 2008 was extremely positive with respect to prior art. •Only „category A‟ documents that related directly to background was cited. European Examiner provided positive comments claiming the invention is novel and inventive. •Patent application has been nationalised in Europe, US, Canada, India, Australia and Japan Brain tumour treatment: Enhanced drug delivery to human Glioblastoma multiforme brain tumour inoculated into athymic mice. Claudin-5 siRNA treated mice on the left shows massively enhanced delivery of small molecules to neuronal environment •There are no encumbrances. The opportunity Retinopathy treatment Localised and inducible modulation of the bloodretina barrier using adeno-associated virus (AAV) for site specific delivery to neuronal tissues. Researchers: Contact: Ref: This technology is currently at the product development stage. Funding from the Wellcome Trust, Science Foundation Ireland, US Department of Defense, Enterprise Ireland, Health Research Board, Fighting Blindness. A campus company „AvenaTherapeutics‟ aims to develop to commercialise the this platform technology Opportunities for investors and/or industry partnership exist. Please contact us if you are interested in forming or becoming part of a team to develop and commercialise this exciting, high growth potential technology Prof. Pete Humphries & Dr. Matt Campbell et al Dr. Emily Vereker, TTO Case Manager [email protected] PH01-112-01 + 353 1 896 4152