Materials and methods for treatment of autosomal dominant cone-rod dystrophy

US10995328B2 · kind B2 · utility

Assignees

• CRISPR THERAPEUTICS AG · CH
• BAYER HEALTHCARE LLC · DE

Inventors

• Ryo Takeuchi · Cambridge, US
• Akiko Noma · Cambridge, US
• Shuying He · Cambridge, US
• Abraham Scaria · Framingham, US

Key dates

• Filing date: Jun 12, 2020
• Grant date: May 4, 2021
• Expiry date: Jun 12, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2800/80
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present application provides materials and methods for treating a patient with autosomal dominant CORD, both ex vivo and in vivo; materials and methods for editing a GUCY2D gene in a human cell; and materials and methods for editing a R838H, R838C, or R838S mutation in a GUCY2D gene in a human cell. The present application also provides one or more gRNAs or sgRNAs for editing a GUCY2D gene; one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene; and a therapeutic comprising at least one or more gRNAs or sgRNAs for editing a R838H, R838C, or R838S mutation in a GUCY2D gene. The present application provides a therapeutic for treating a patient with autosomal dominant CORD. The present application also provides a kit for treating a patient with autosomal dominant CORD. In addition, the present application provides a self-inactivating CRISPR-Cas system.