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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