/Highly Efficient Transduction And Lateral Spread In The Retina By A Novel Aav Virus Enhanced By Rational Design
Abstract
rAAV particles comprising a new capsid variant, AAV44.9(E531D) are described herein, as well as a polynucleotide encoding same, and the use of the rAAV particles in the treatment of the eye, including treatment of retinal disorders. The disclosure also shows that the rAAV particles comprising an AAV44.9(E531D) capsid exhibit enhanced lateral spread after subretinal injection to a fovea of the subject, wherein detachment of the fovea is minimized. Methods of treatment comprising administering rAAV particles to a mammal in need thereof, and methods of transducing photoreceptor and RPE cells with rAAV particles, are also provided.
Full Text
What is claimed is:
rAAV particles comprising a new capsid variant, AAV44.9(E531D) are described herein, as well as a polynucleotide encoding same, and the use of the rAAV particles in the treatment of the eye, including treatment of retinal disorders. The disclosure also shows that the rAAV particles comprising an AAV44.9(E531D) capsid exhibit enhanced lateral spread after subretinal injection to a fovea of the subject, wherein detachment of the fovea is minimized. Methods of treatment comprising administering rAAV particles to a mammal in need thereof, and methods of transducing photoreceptor and RPE cells with rAAV particles, are also provided.
Timeline
Filed
05/08/2026Published
09/03/2026Granted
Not AvailableIPC Codes(3)
A61K 48/00:Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/005:from viruses
C12N 15/86:Viral vectors