Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems

US12410442B2 · kind B2 · utility

Assignees

• REGENERON PHARMACEUTICALS, INC. · US
• Decibel Therapeutics, Inc. · US

Inventors

• Joseph BURNS · Newton, US
• Kathryn ELLIS · Arlington, US
• Adam PALERMO · Boston, US
• Martin SCHWANDER · Riverview, US
• Jonathon WHITTON · Cambridge, US
• Leah Sabin · Goldens Bridge, US
• Christos Kyratsous · Dobbs Ferry, US
• Meghan Drummond Samuelson · Tarrytown, US

Key dates

• Filing date: Aug 6, 2021
• Grant date: Sep 9, 2025
• Expiry date: Apr 8, 2044

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2840/44
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The disclosure features compositions and methods for the treatment of sensorineural hearing loss and auditory neuropathy, particularly forms of the disease that are associated with mutations in otoferlin (OTOF), by way of OTOF gene therapy. The disclosure provides a variety of compositions that include a first nucleic acid vector that contains a polynucleotide encoding an N-terminal portion of an OTOF protein and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF protein. These vectors can be used to increase the expression of OTOF in a subject, such as a human subject suffering from sensorineural hearing loss.