IP Library › Granted Patent US 12,252,520
Granted Patent B2
US 12,252,520 · App. 17/395,773 · Granted Mar 18, 2025

Myosin 15 promoters and uses thereof

Inventors: Joseph Burns (Newton, MA); Kathryn Ellis (Arlington, MA); Adam Palermo (Somerville, MA); Martin Schwander (Auburndale, MA); Jonathon Whitton (Cambridge, MA); Leah Sabin (Goldens Bridge, NY); Christos Kyratsous (Irvington, NY); Meghan Drummond Samuelson (Katonah, NY)
Assignees: Decibel Therapeutics, Inc.; Regeneron Pharmaceuticals, Inc.
C07K14/4716A61K48/0058C12N15/86A61K38/00C12N2750/14143
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Quick Facts
Patent No.
US 12,252,520
App. No.
17/395,773
Granted
Mar 18, 2025
Kind
B2
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.

Claims (13)

1. A polynucleotide having at least 85% sequence identity to SEQ ID NO: 15.

2. A nucleic acid vector comprising the polynucleotide of claim 1 .

3. The nucleic acid vector of claim 2 , wherein the polynucleotide is operably linked to a transgene.

4. The nucleic acid vector of claim 3 , wherein the transgene comprises a nucleic acid sequence encoding a therapeutic protein.

5. The nucleic acid vector of claim 4 , wherein the therapeutic protein is encoded by a gene selected from the group consisting of ACTG1, FSCN2, RDX, POU4F3, TRIOBP, TPRN, XIRP2, ATOH1, GFI1, CHRNA9, CIB3, CDH23, PCDH15, KNCN, DFNB59, OTOF, MKRN2OS, LHX3, TMC1, MYO15, MYO7A, MYO6, MYO3A, MYO3B, GRXCR1, PTPRQ, LCE6A, LOXHD1, ART1, ATP2B2, CIB2, CACNA2D4, CABP2, EPS8, EPS8L2, ESPN, ESPNL, PRPH2, STRC, SLC8A2, ZCCHC12, LRTOMT2, LRTOMT1, USH1C, ELFN1, TTC24, DYTN, KCP, CCER2, LRTM2, KCNA10, NTF3, CLRN1, CLRN2, SKOR1, TCTEX1D1, FCRLB, SLC17A8, GRXCR2, BDNF, SERPINE3, NHLH1, HSP70, HSP90, ATF6, PERK, IRE1, and BIP.

6. A method of expressing a therapeutic protein in a mammalian hair cell, comprising contacting the mammalian hair cell with the nucleic acid vector of claim 4 .

7. A method of treating a subject having or at risk of developing hearing loss, comprising administering to the subject an effective amount of the nucleic acid vector of claim 3 .

8. A method of treating a subject having or at risk of developing vestibular dysfunction, comprising administering to the subject an effective amount of the nucleic acid vector of claim 3 .

9. A method of promoting hair cell regeneration in a subject in need thereof, comprising administering to the subject an effective amount of the nucleic acid vector of claim 3 .

10. A method of treating a subject having tinnitus, comprising administering to the subject an effective amount of the nucleic acid vector of claim 3 .

11. The nucleic acid vector of claim 2 , wherein the vector is an adeno-associated virus vector.

12. The polynucleotide of claim 1 , wherein the polynucleotide has at least 95% sequence identity to SEQ ID NO: 15.

13. The polynucleotide of claim 12 , wherein the polynucleotide has the sequence of SEQ ID NO: 15.

Assignments (6)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 20, 2023
From: SABIN, LEAH; KYRATSOUS, CHRISTOS; DRUMMOND SAMUELSON, MEGHAN
To: REGENERON PHARMACEUTICALS, INC.
Reel/Frame 064325/0167 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 4, 2022
From: SCHWANDER, MARTIN
To: DECIBEL THERAPEUTICS, INC.
Reel/Frame 059169/0488 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 4, 2022
From: PALERMO, ADAM
To: DECIBEL THERAPEUTICS, INC.
Reel/Frame 059169/0404 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 4, 2022
From: WHITTON, JONATHON
To: DECIBEL THERAPEUTICS, INC.
Reel/Frame 059169/0512 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 4, 2022
From: BURNS, JOSEPH
To: DECIBEL THERAPEUTICS, INC.
Reel/Frame 059169/0757 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 4, 2022
From: ELLIS, KATHRYN
To: DECIBEL THERAPEUTICS, INC.
Reel/Frame 059170/0215 →
Continuity (5)
Continuation PCTUS2020017292 · Feb 7, 2020
Provisional Application 62965773 · Jan 24, 2020
Provisional Application 62928311 · Oct 30, 2019
Provisional Application 62802874 · Feb 8, 2019
Related Publication 20210388045A1 · Dec 16, 2021
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Cited By (1)
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