Otoferlin Dual Vector Systems for Treating Sensorineural Hearing Loss

ChangeBridge: Patent Grants - Peptides (C07K)

Published March 31st, 2026

Detected March 31st, 2026

Summary

The USPTO granted Regeneron Pharmaceuticals Patent US12589168B2 for compositions and methods treating sensorineural hearing loss using otoferlin (OTOF) dual vector gene therapy systems. The patent covers a two-vector approach delivering N-terminal and C-terminal portions of OTOF isoform 5 to restore gene function in patients with OTOF mutations. Application No. 17733744 was filed April 29, 2022.

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What changed

The USPTO issued Patent US12589168B2 to Regeneron Pharmaceuticals covering dual nucleic acid vector compositions for OTOF gene therapy to treat sensorineural hearing loss and auditory neuropathy. The patent claims a first vector encoding an N-terminal OTOF isoform 5 portion and a second vector encoding a C-terminal portion, designed to overcome gene size limitations in adeno-associated virus (AAV) delivery systems. Key inventors include Joseph Burns, Kathryn Ellis, and others. The patent is classified under C07K 14/4716 and A61P 27/16.

Patent holders and licensees developing OTOF gene therapies for hearing loss should review this granted patent to assess freedom-to-operate considerations and potential licensing requirements. Generic drug and biosimilar manufacturers should note that Regeneron now holds enforceable intellectual property rights covering split otoferlin vector approaches for this therapeutic indication. No immediate compliance actions are required for entities not currently operating in this gene therapy space.

Source document (simplified)

← USPTO Patent Grants

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

Grant US12589168B2 Kind: B2 Mar 31, 2026

Assignee

Regeneron Pharmaceuticals, Inc.

Inventors

Joseph Burns, Kathryn Ellis, Tyler Gibson, Adam Palermo, Martin Schwander, Jonathon Whitton, Leah Sabin, Christos Kyratsous, Meghan Drummond Samuelson

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 a mutation 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 isoform 5 protein and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF isoform 5 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.