Ceregene, Inc.

Adverum Biotechnologies, Inc.

Mehdi Gasmi

Annahita Keravala

Raymond T Bartus

Thomas W Chalberg, Jr

Christopher Chavez

Mark Blumenkranz

Kathie M Bishop

Elias T Ketchum

Dominick A Vacante

Diana Cepeda

Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins. Also provided are compositions and methods for the use of the recombinant AAV virions, such as for the treatment or prophylaxis of a disease or disorder.

Variant AAV capsids for intravitreal delivery

Provided are mutant viral capsid cell libraries, individual cells of such libraries, systems, vectors, and methods for generating the cell libraries, and methods of use thereof to screen for mutant viral capsids with desired characteristics.

Mutant viral capsid libraries and related systems and methods

The invention provides methods for treating ocular diseases using a recombinant vehicle to express a protein useful in the treatment of ocular disease, with particular preference for use of neurotrophin-4 (NT4) for targeting subpopulations of cells in the retina. A genetically engineered gene transfer vector containing sequences encoding a growth factor such as neurotrophin-4 (NT4) is used to transduce cells of the retinal ganglion cell (RGC) layer, in situ, via administration of the vector intravitreally. Accordingly, methods are disclosed for treating subjects in need thereof by therapeutic protein delivery via a recombinant expression vector, including rescue of photoreceptors by targeting the RGC layer subpopulation of retinal cells.

Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

The needle assembly of the invention is a quad-partite system for use with an arc-center stereotactic headframe that allows a clinician to deliver recombinant expression vectors through the needle assembly to targeted sites at exact depths in the brain. Methods for use of the needle assembly with a rube insert for the needle cannula that does not bind proteins, as in protein viral capsids, permit delivery of precise volumes of pharmaceutical compositions containing viral recombinant expression vectors for gene therapy.

Needle assembly for use in delivering precise dosages of proteinaceous pharmaceutical compositions and methods for use of same

The needle assembly of the invention is a quad-partite system for use with an arc-center stereotactic headframe that allows a clinician to deliver recombinant expression vectors through the needle assembly to targeted sites at exact depths in the brain. Methods for use of the needle assembly with a tube insert for the needle cannula that does not bind proteins, as in protein viral capsids, permit delivery of precise volumes of pharmaceutical compositions containing viral recombinant expression vectors for gene therapy.

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San Diego, CA, United States of America

Mehdi Gasmi