Trans-splicing molecules

US11993776B2 · kind B2 · utility

Assignees

• Ascidian Therapeutics, Inc. · US
• The Trustees of the University of Pennsylvania · US

Inventors

• Philip R. Johnson · Lansdowne, US
• Bruce C. Schnepp · Havertown, US
• Jean Bennett · Via Enrico Fermi, US
• Scott J. Dooley · Boston, US
• Krishna J. Fisher · New Orleans, US
• Junwei Sun · Philadelphia, US

Key dates

• Filing date: Apr 17, 2019
• Grant date: May 28, 2024
• Expiry date: Jul 12, 2040

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/34
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.