Decibel Therapeutics, Inc.

Regeneron Pharmaceuticals, Inc.

Martin Schwander

Kathryn Ellis

Jonathon Whitton

Joseph Burns

Adam Palermo

Christos Kyratsous

Meghan Drummond Samuelson

Leah Sabin

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.

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

The disclosure provides polynucleotides containing regions of the Myosin 15 (Myo15) promoter, as well as vectors containing the same, that can be used to promote expression of a transgene specifically in hair cells. The polynucleotides described herein may be operably linked to a transgene, such as a transgene encoding a therapeutic protein, so as to promote hair cell-specific expression of the transgene. The polynucleotides described herein may be operably linked to a therapeutic transgene and used for the treatment of subjects having or at risk of developing hearing loss or vestibular dysfunction.

Myosin 15 promoters and uses thereof

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Auburndale, MA, United States of America

Martin Schwander