Kathryn Eve Ellis

US Patent:

20210236654, Aug 5, 2021

Filed:

Apr 26, 2019

Appl. No.:

17/050741

Inventors:

- Boston MA, US
Kathryn ELLIS - Arlington MA, US
Adam PALERMO - Somerville MA, US
Martin SCHWANDER - Auburndale MA, US
Jonathon WHITTON - Cambridge MA, US

International Classification:

A61K 48/00
C07K 14/47
A61P 27/16

Abstract:

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.

US Patent:

20200155705, May 21, 2020

Filed:

Apr 26, 2019

Appl. No.:

16/395999

Inventors:

- Boston MA, US
Kathryn ELLIS - Arlington MA, US
Adam PALERMO - Cambridge MA, US
Martin SCHWANDER - Auburndale MA, US
Jonathon WHITTON - Cambridge MA, US

International Classification:

A61K 48/00
C07K 14/47
C12N 15/86

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.