Abstract: 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.
Type:
Grant
Filed:
April 24, 2020
Date of Patent:
November 29, 2022
Assignee:
ADVERUM BIOTECHNOLOGIES, INC.
Inventors:
Annahita Keravala, Diana Cepeda, Mehdi Gasmi
Abstract: 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.
Type:
Grant
Filed:
January 23, 2020
Date of Patent:
August 30, 2022
Assignee:
Adverum Biotechnologies, Inc.
Inventors:
Christopher Chavez, Mehdi Gasmi, Annahita Keravala, Thomas W. Chalberg
Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Abstract: Methods and compositions are provided for intravitreally delivering a polynucleotide to cone photoreceptors. Aspects of the methods include injecting a recombinant adeno-associated virus comprising a polynucleotide of interest into the vitreous of the eye. These methods and compositions find particular use in treating ocular disorders associated with cone dysfunction and/or death.
Type:
Grant
Filed:
March 2, 2016
Date of Patent:
June 1, 2021
Assignees:
Adverum Biotechnologies, Inc., University of Washington
Inventors:
Thomas W. Chalberg, Jr., Jay Neitz, Maureen Neitz
Abstract: 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.
Type:
Grant
Filed:
December 22, 2016
Date of Patent:
March 10, 2020
Assignee:
Adverum Biotechnologies, Inc.
Inventors:
Christopher Chavez, Mehdi Gasmi, Annahita Keravala, Thomas W. Chalberg
Abstract: A vector comprising a promoter operably linked to a nucleic acid sequence encoding human aldehyde dehydrogenase, as well as a composition comprising the vector and method of using the vector to treat aldehyde dehydrogenase deficiency, or to prevent or treat a disease characterized by aldehyde dehydrogenase deficiency.
Type:
Application
Filed:
July 26, 2017
Publication date:
May 30, 2019
Applicants:
Cornell University, Adverum Biotechnologies, Inc.
Inventors:
Mehdi Gasmi, Ronald G. Crystal, Odelya E. Pagovich