Compositions and methods for treatment of dominant retinitis pigmentosa

US12203074B2 · kind B2 · utility

Assignees

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

Inventors

• Alfred S. Lewin · Gainesville, US
• William W. Hauswirth · Gainesville, US
• Michael T. Massengill · Gainesville, US
• William A. Beltran · Philadelphia, US
• Gustavo D. Aguirre · Philadelphia, US
• Artur Cideciyan · Philadelphia, US
• Samuel G. Jacobson · Levittown, US

Key dates

• Filing date: Jun 3, 2019
• Grant date: Jan 21, 2025
• Expiry date: Feb 11, 2042

Classification

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

Abstract

Aspects of the disclosure relate to methods and compositions useful for treating retinitis pigmentosa. In some aspects, the disclosure provides compositions and methods for delivering an interfering RNA to a subject in order to reduce expression of one or both alleles of an endogenous RHO gene (for example a mutant rho allele associated with retinitis pigmentosa) in a subject. In some embodiments, a replacement RHO coding sequence that is resistant to the interfering RNA also is delivered to the subject.