Adeno-associated virus virions with variant capsids and methods of use thereof

US12031147B2 · kind B2 · utility

Assignees

• The Regents of the University of California · US
• Novartis AG · CH

Inventors

• John G. Flannery · Berkeley, US
• Scott F. Geller · Berkeley, US
• Karen I. Guerin · Braintree, US

Key dates

• Filing date: May 27, 2021
• Grant date: Jul 9, 2024
• Expiry date: Feb 25, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14145
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The present disclosure provides recombinant adeno-associated virus (AAV) virions comprising: a) a variant capsid protein; and b) a heterologous nucleic acid comprising one or more nucleotide sequences encoding one or more heterologous gene products. The rAAV virions are useful for delivery of gene products to a retinal cell. The present disclosure provides methods of delivering a gene product to a retinal cell in an individual.