IP Library › Granted Patent US 12,391,936
Granted Patent B2
US 12,391,936 · App. 16/783,148 · Granted Aug 19, 2025

Gene therapy systems and related methods for treatment of hearing loss

Inventors: Hinrich Staecker (Leawood, KS); Caesar James Ayala (Gainesville, FL)
Assignee: Rescue Hearing, Inc.
C12N9/6421A61K9/0019A61K9/0046A61K48/0058A61N1/0541A61N1/36038C12N5/0696C12N15/86
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Quick Facts
Patent No.
US 12,391,936
App. No.
16/783,148
Granted
Aug 19, 2025
Kind
B2
Abstract

The present disclosure describes gene therapy systems, and related methods, useful for treating and/or preventing deafness caused by genetic mutation of the TMPRSS3 gene or the LOXHD1 gene. The compositions and methods disclosed herein use adeno-associated viral (AAV) vector gene delivery of TRMPSS3 or LOXHD1 into the inner ear to restore activity of the TMPRSS3 gene or the LOXHD1 gene, respectively, promote hair cell survival and restore hearing in patients suffering from hearing loss. As disclosed herein, the systems and methods may utilize a combination of gene therapy (e.g., molecular therapeutics) for hearing loss caused by a genetic mutation together with implantation of a cochlear implant.

Claims (17)

1. A method for treating or preventing hearing loss in a subject in need thereof, comprising the steps of:

administering to the subject an effective amount of an expression vector to result in TMPRSS3 expression in inner and outer hair cells and spiral ganglion cells, the expression vector comprising

an initial AAV2 inverted terminating repeat (ITR) sequence,

an enhancer,

a nucleic acid sequence having 100% sequence identity to the nucleic acid of SEQ ID NO: 1 or SEQ ID NO: 3,

a promoter operatively linked to the nucleic acid sequence, wherein the promoter is a human cytomegalovirus (hCMV) promoter,

a bGH poly(A) signal, and

a closing AAV2 inverted terminating repeat (ITR) sequence,

wherein the expression vector is a wildtype AAV2 adeno-associated viral vector; and

implanting a cochlear implant in the subject.

2. The method of claim 1 , wherein the administration of the expression vector is performed prior to the implantation of the cochlear implant.

3. The method of claim 1 , wherein the administration of the expression vector is performed subsequent to the implantation of the cochlear implant.

4. The method of claim 1 , wherein the administration of the expression vector and the cochlear implant are performed concurrently.

5. The method of claim 1 , wherein the expression vector is administered by injection into the inner ear of the subject.

6. The method of claim 5 , wherein the injection method is selected from the group consisting of cochleostomy, round window membrane, endolymphatic sac, scala media, canalostomy, scala media via the endolymphatic sac, or any combination thereof.

7. The method of claim 1 , wherein the subject has one or more genetic risk factors associated with hearing loss.

8. The method of claim 7 , wherein one of the genetic risk factors is selected from the group consisting of a mutation in the TMPRSS3 gene or a mutation in the LOXHD1 gene.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 20, 2025
From: STAECKER, HINRICH; AYALA, CAESAR JAMES
To: RESCUE HEARING, INC.
Reel/Frame 071171/0579 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 1, 2022
From: STAECKER, HINRICH; AYALA, CAESAR JAMES
To: RESCUE HEARING INC
Reel/Frame 058848/0148 →
Continuity (5)
Continuation In Part 16726495 · Dec 24, 2019
Continuation In Part 16488103
Provisional Application 62472790 · Mar 17, 2017
Provisional Application 62531522 · Jul 12, 2017
Related Publication 20200181596A1 · Jun 11, 2020
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