"A woman from Oxford has become the first person in the world to have gene therapy to try to halt the most common form of blindness in the Western world"
"The treatment was carried out under local anaesthetic last month at Oxford Eye Hospital by Robert MacLaren, professor of ophthalmology at the University of Oxford."
Benitec approach -
Age-Related Macular Degeneration (AMD)The DiseaseAMD accounts for 8% of blindness worldwide and has been projected to impact up to 196 million patients by 2020 and up to 288 million by 2040.The wet form of the disease accounts for about 10% of all AMD patients but accounts for up to 90% of all the blindness.Wet AMD is characterised by the growth of new blood vessels into the eye, a phenomenon that has been associated with the expression of abnormally high levels of proteins from the vascular endothelial growth factor (VEGF) family.The most commonly used standard of care treatments for AMD require an intravitreal injection into the eye as frequently as monthly or bi-monthly. These frequent injections may be required indefinitely to be able to halt progression of the disease and stabilise vision. The Benitec ApproachOur AMD product candidate, BB-201, is comprised of a novel adeno associated virus capsid (AAV) and a recombinant DNA cassette, engineered to express steady state levels of three short hairpin RNA that inhibit VEGF-a, VEGF-b and PlGF, three clinically validated targets whose expression is shown to lead to the progression of AMD.Along with our collaborators, we have identified novel AAV capsids for delivery to key cell layers within the retina using direct intravitreal injection, a commercially attractive route of administration.The AMD program is our first program in this space and we anticipate being able to build a ddRNAi franchise for other retinal diseases using the same delivery system.
BLT Price at posting:
12.3¢ Sentiment: Buy Disclosure: Held
