Regeneron Pharmaceuticals, Inc.

Decibel Therapeutics, Inc.

Regeneran Pharmaceuticals, Inc.

Leah Sabin

Andrew J Murphy

Aris N Economides

Christos Kyratsous

Brian Zambrowicz

Lori C Morton

Chia-Jen Siao

Cheng Wang

Meghan Drummond Samuelson

Dan Chalothorn

Christopher Schoenherr

KehDih Lai

Adam Palermo

Kathryn Ellis

Martin Schwander

Jonathon Whitton

Joseph Burns

Rachel Sattler

Qing Fang

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

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized albumin (ALB) locus and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized albumin locus express a human albumin protein or a chimeric albumin protein, fragments of which are from human albumin. Methods are provided for using such non-human animals comprising a humanized albumin locus to assess in vivo efficacy of human-albumin-targeting reagents such as nuclease agents designed to target human albumin.

Method of using a genetically modified mouse that expresses human albumin

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

Genetically modified mouse that expresses human albumin

Growing community of inventors

Goldens Bridge, NY, United States of America

Leah Sabin