Hearing Loss Gene Therapy Registry

An overview of all human hearing loss gene therapy trials that outlines:

the target population
what the therapy is made of
the delivery method to the inner ear
the supporting evidence

All therapies listed here are investigational. Inclusion does not mean a treatment is approved, available, safe, or effective.

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Clinical programs

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OTOF programsGJB2 programs

Human hearing loss gene therapy programs, including countries, vector, promoter, fenestration, study status, start date, and FDA approval.
Program	Gene	Countries	Vector	Promoter	Fenestration	Status	Study start	FDA
DB-OTO (CHORD)Regeneron Pharmaceuticals	OTOF	Germany, Japan, Spain, United Kingdom, and United States	Dual AAV1 vectors reconstituting full-length human OTOF transcript variant 5 (hOTOFv5)	mMyo15, a synthetic hair-cell-specific promoter derived from murine Myosin 15a regulatory sequence	Yes — lateral semicircular canal (LSCC)	Actively Recruiting	Jun 27, 2023	Yes
AK-OTOF-101Akouos, Inc. / Eli Lilly	OTOF	Canada, Taiwan, United Kingdom, and United States	Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA	Ubiquitous promoter; specific identity is not publicly disclosed	Yes — stapes footplate vent (fenestration)	Actively Recruiting	Sep 15, 2023	No
SENS-501 (Audiogene)Sensorion	OTOF	Australia and France	Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution	CMV promoter	Yes — stapes footplate (stapedotomy)	Active, Not Recruiting	Jun 21, 2024	No
OTOV101N + OTOV101COtovia Therapeutics	OTOF	China	Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution	mMyo15 hair-cell-specific promoter	No separate fenestration reported; round-window membrane injection	Active	Jun 26, 2023	No
EH002Yilai Shu / Eye & ENT Hospital of Fudan University	OTOF	China	OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed	Not publicly disclosed	Not publicly disclosed	Actively Recruiting	Nov 22, 2024	No
SKY-GJB2 (SONIX)Skylark Bio Inc.	GJB2	United States	AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed	Not publicly disclosed	No additional fenestration reported	Actively Recruiting	May 28, 2026	No

DB-OTO (CHORD)

Regeneron Pharmaceuticals

OTOF

Countries: Germany, Japan, Spain, United Kingdom, and United States
Vector: Dual AAV1 vectors reconstituting full-length human OTOF transcript variant 5 (hOTOFv5)
Promoter: mMyo15, a synthetic hair-cell-specific promoter derived from murine Myosin 15a regulatory sequence
Fenestration: Yes — lateral semicircular canal (LSCC)
Study status: Actively Recruiting
Study start date: Jun 27, 2023
FDA approval: Yes

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AK-OTOF-101

Akouos, Inc. / Eli Lilly

OTOF

Countries: Canada, Taiwan, United Kingdom, and United States
Vector: Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA
Promoter: Ubiquitous promoter; specific identity is not publicly disclosed
Fenestration: Yes — stapes footplate vent (fenestration)
Study status: Actively Recruiting
Study start date: Sep 15, 2023
FDA approval: No

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SENS-501 (Audiogene)

Sensorion

OTOF

Countries: Australia and France
Vector: Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution
Promoter: CMV promoter
Fenestration: Yes — stapes footplate (stapedotomy)
Study status: Active, Not Recruiting
Study start date: Jun 21, 2024
FDA approval: No

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OTOV101N + OTOV101C

Otovia Therapeutics

OTOF

Countries: China
Vector: Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution
Promoter: mMyo15 hair-cell-specific promoter
Fenestration: No separate fenestration reported; round-window membrane injection
Study status: Active
Study start date: Jun 26, 2023
FDA approval: No

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EH002

Yilai Shu / Eye & ENT Hospital of Fudan University

OTOF

Countries: China
Vector: OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed
Promoter: Not publicly disclosed
Fenestration: Not publicly disclosed
Study status: Actively Recruiting
Study start date: Nov 22, 2024
FDA approval: No

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SKY-GJB2 (SONIX)

Skylark Bio Inc.

GJB2

Countries: United States
Vector: AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed
Promoter: Not publicly disclosed
Fenestration: No additional fenestration reported
Study status: Actively Recruiting
Study start date: May 28, 2026
FDA approval: No

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Evidence Methodology

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Study status and eligibility: The official ClinicalTrials.gov record
Therapy design and delivery: Peer-reviewed publications + other technical sources

Note: any information that was not discoverable publicly is marked as "Not publicly disclosed."

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