Title:
COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS USING OTOFERLIN DUAL VECTOR SYSTEMS
Document Type and Number:
WIPO Patent Application WO/2021/087296
Kind Code:
A8
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 OTOE 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.
Inventors:
BURNS JOSEPH (US)
ELLIS KATHRYN (US)
GIBSON TYLER (US)
PALERMO ADAM (US)
SCHWANDER MARTIN (US)
WHITTON JONATHON (US)
SABIN LEAH (US)
KYRATSOUS CHRISTOS (US)
DRUMMOND MEGHAN (US)
ELLIS KATHRYN (US)
GIBSON TYLER (US)
PALERMO ADAM (US)
SCHWANDER MARTIN (US)
WHITTON JONATHON (US)
SABIN LEAH (US)
KYRATSOUS CHRISTOS (US)
DRUMMOND MEGHAN (US)
Application Number:
PCT/US2020/058265
Publication Date:
August 05, 2021
Filing Date:
October 30, 2020
Export Citation:
Assignee:
DECIBEL THERAPEUTICS INC (US)
REGENERON PHARMA (US)
REGENERON PHARMA (US)
International Classes:
C07K14/47; C12N15/63; C12N15/86
Attorney, Agent or Firm:
BELLIVEAU, Michael, J. (US)
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