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AssigneeDecibel Therapeutics, Inc.
InventorsQi-Ying HU, Orion Paul Keifer, JR., Seth Koehler, John Lee, Adam Palermo, Ning Pan, Jonathan B. Sellon, Jonathon Whitton
Estimating the term…
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 protein (e.g., an OTOF isoform 5 protein) and a second nucleic acid vector that contains a polynucleotide encoding a C-terminal portion of an OTOF protein (e.g., 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.
A method of improving hearing in treating a human subject having biallelic otoferlin (OTOF) mutations and profound sensorineural hearing loss, the method comprising administering to an inner ear of the subject an amount of 1×1013 vg/mL to 1×1014 vg/mL of an OTOF dual vector system in a volume of 200-250 μL by intracochlear injection, wherein the OTOF dual vector system comprises:
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An aqueous suspension comprising an OTOF dual vector system, 10 mM sodium phosphate or disodium phosphate, 180 mM sodium chloride, 5% (w/v) sucrose, and 0.001% (w/v) poloxamer 188 at a pH of 7.4, wherein the OTOF dual vector system comprises:
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Dependencies are inferred from the claim text.