AAV-mediated gene therapy for RPGR X-linked retinal degeneration

US10383922B2 · kind B2 · utility

Assignees

• The Trustees of the University of Pennsylvania · US
• University of Florida Research Foundation, INC. · US

Inventors

• William A. Beltran · Philadelphia, US
• Gustavo D. Aguirre · Philadelphia, US
• Samuel G. Jacobson · Levittown, US
• Artur Cideciyan · Philadelphia, US
• Alfred S. Lewin · Gainesville, US
• Sanford L. Boye · Gainesville, US
• William W. Hauswirth · Gainesville, US
• Wen-Tao Deng · Suzhou, CN

Key dates

• Filing date: Sep 8, 2017
• Grant date: Aug 20, 2019
• Expiry date: Sep 8, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y306/05
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to the subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.