Compositions and methods for correction of heritable ocular disease

US10987433B2 · kind B2 · utility

Assignee

• The Trustees of the University of Pennsylvania · US

Inventors

• Jean Bennett · Via Enrico Fermi, US
• Jeannette Bennicelli · Philadelphia, US
• Scott J. Dooley · Boston, US
• Lloyd G. Mitchell · Durham, US

Key dates

• Filing date: Nov 18, 2016
• Grant date: Apr 27, 2021
• Expiry date: Mar 21, 2037

Classification

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

Abstract

A nucleic acid trans-splicing molecule is provided that can replace an exon in a targeted mammalian ocular gene carrying a defect or mutation causing an ocular disease with an exon having the naturally-occurring sequence without the defect or mutation. A method of treating an ocular disease, e.g., Stargardt's Disease, caused by a defect or mutation in a target gene, e.g., ABCA4 comprising: administering to the ocular cells of a subject having an ocular disease a composition comprising a recombinant AAV comprising a nucleic acid trans-splicing molecule as described above.