Composition and methods for highly efficient gene transfer using AAV capsid variants

US9909142B2 · kind B2 · utility

Assignee

• THE CHILDREN'S HOSPITAL OF PHILADELPHIA · US

Inventors

• Mustafa Yazicioglu · Kandıra, TR
• Federico Mingozzi · Paris, FR
• Xavier Anguela · Philadelphia, US
• Katherine A. High · Yeadon, US

Key dates

• Filing date: Apr 18, 2013
• Grant date: Mar 6, 2018
• Expiry date: Apr 18, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.