Hearing-loss gene therapy studies

Compare the study populations, investigational treatments, and delivery methods. There may be inaccuracies on this page. Always double check important information and if needed, please submit a correction.

Dataset reviewed throughAug 11, 2026

Sources

Terminology Dictionary

Target gene: The gene the treatment is designed to address.
Gene replacement: Delivery of a working gene copy intended to restore a missing or impaired function.
Vector / payload: The delivery vehicle and the genetic material it carries.
Promoter: A DNA control sequence that helps determine where and how strongly the delivered gene is active.
Cochlear access: The route used to reach the inner ear and administer the treatment.
Laterality: Whether treatment is given to one ear or both ears.
Phase 1 / 2: An early human study designed mainly to examine safety, dosing, and initial signs of biological or clinical effect.
Early clinical: An early human study for which a formal clinical phase is not assigned or publicly stated.
Biallelic: A disease-causing change is present in both copies of a gene.
Dual vector: Two delivery vehicles carry separate parts of a gene that is too large to fit into one vehicle.
Intracochlear: Delivered into the fluid-filled inner-ear structure involved in hearing.

Search all fieldsTarget geneStudy statusAge groupSort by

6 of 6 programs shown

Human hearing-loss gene therapy program comparison
Program	Phase / status	Gene	Countries	Target Population	Vector	Promoter	Surgical Approach	Study start	FDA approval	Sources
AK-OTOF-101 Akouos, Inc. / Eli Lilly	Phase 1/2 Actively Recruiting	OTOF	Canada, Taiwan, United Kingdom, and United States	People with OTOF-mediated sensorineural hearing loss Cohort 1a: 7–17 yearsCohort 1b: first 3 participants are 2 years and older, next 3 may be any ageCohort 2: first 3 participants are 2 years and older, next 3 may be any agebilateral expansion: any age	Dual AAVAnc80 vectors carrying 5′ and 3′ components of approximately 6 kb human OTOF cDNA	Ubiquitous promoter; specific identity is not publicly disclosed	One-time intracochlear administration using the Akouos delivery device Fenestration: Yes — stapes footplate vent (fenestration) Access: Transcanal tympanotomy to the round-window membrane with a stapes-footplate vent	Sep 15, 2023	No	Registry (opens in a new tab)Sources
DB-OTO (CHORD) Regeneron Pharmaceuticals	Phase 1/2 Actively Recruiting	OTOF	Germany, Japan, Spain, United Kingdom, and United States	Children and infants with biallelic OTOF-related hearing loss Up to 17 years; cohort-specific	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	One-time intracochlear injection through the round-window membrane with lateral semicircular canal fenestration Fenestration: Yes — lateral semicircular canal (LSCC) Access: Round-window membrane injection with lateral semicircular canal fenestration	Jun 27, 2023	YesApr 23,2026Source	Registry (opens in a new tab)Sources
EH002 Yilai Shu / Eye & ENT Hospital of Fudan University	Early clinical Actively Recruiting	OTOF	China	People with DFNB9 congenital hearing loss 6 months and older; additional eligibility criteria apply	OTOF gene therapy with program-specific AAV1 neutralizing-antibody screening; the complete vector cassette and split strategy are not publicly disclosed	Not publicly disclosed	Intracochlear injection Fenestration: Not publicly disclosed Access: The precise cochlear access route is not publicly disclosed	Nov 22, 2024	No	Registry (opens in a new tab)Sources
OTOV101N + OTOV101C Otovia Therapeutics	Early clinical Active	OTOF	China	People aged 1 year and older with OTOF-related hearing loss 1 year and older	Dual Anc80L65 vectors encoding human OTOF, split between exons 20 and 21 with splice-donor/acceptor-mediated reconstitution	mMyo15 hair-cell-specific promoter	Intracochlear administration Fenestration: No separate fenestration reported; round-window membrane injection Access: Round-window membrane injection; preclinical program work used a transmastoid facial-recess corridor	Jun 26, 2023	No	Registry (opens in a new tab)Sources
SENS-501 (Audiogene) Sensorion	Phase 1/2 Active, Not Recruiting	OTOF	Australia and France	Children with severe-to-profound pre-lingual OTOF-related hearing loss 6–31 months	Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution	CMV promoter	Unilateral intracochlear injection with a proprietary delivery device Fenestration: Yes — stapes footplate (stapedotomy) Access: Round-window membrane injection; translational work describes stapedotomy pressure relief	Jun 21, 2024	No	Registry (opens in a new tab)Sources
SKY-GJB2 (SONIX) Skylark Bio Inc.	Phase 1/2 Actively Recruiting	GJB2	United States	Children with biallelic GJB2-mediated hearing loss 9 months–7 years	AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed	Not publicly disclosed	Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT Fenestration: No additional fenestration reported Access: Round-window access via transcanal endoscopic tympanotomy	May 28, 2026	No	Registry (opens in a new tab)Sources

AK-OTOF-101

Akouos, Inc. / Eli Lilly

OTOF

Phase 1/2Actively Recruiting

Who can take part: People with OTOF-mediated sensorineural hearing loss · Cohort 1a: 7–17 years Cohort 1b: first 3 participants are 2 years and older, next 3 may be any age Cohort 2: first 3 participants are 2 years and older, next 3 may be any age bilateral expansion: any age
Countries: Canada, Taiwan, United Kingdom, and United States
Study start date: Sep 15, 2023
FDA approval: No
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
Surgical Approach: One-time intracochlear administration using the Akouos delivery device Fenestration: Yes — stapes footplate vent (fenestration) Cochlear access: Transcanal tympanotomy to the round-window membrane with a stapes-footplate vent Unilateral cohorts; bilateral expansion

Full study record View sources

DB-OTO (CHORD)

Regeneron Pharmaceuticals

OTOF

Phase 1/2Actively Recruiting

Who can take part: Children and infants with biallelic OTOF-related hearing loss · Up to 17 years; cohort-specific
Countries: Germany, Japan, Spain, United Kingdom, and United States
Study start date: Jun 27, 2023
FDA approval: YesApr 23,2026Source
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
Surgical Approach: One-time intracochlear injection through the round-window membrane with lateral semicircular canal fenestration Fenestration: Yes — lateral semicircular canal (LSCC) Cochlear access: Round-window membrane injection with lateral semicircular canal fenestration Unilateral dose escalation; bilateral expansion

Full study record View sources

EH002

Yilai Shu / Eye & ENT Hospital of Fudan University

OTOF

Early clinicalActively Recruiting

Who can take part: People with DFNB9 congenital hearing loss · 6 months and older; additional eligibility criteria apply
Countries: China
Study start date: Nov 22, 2024
FDA approval: No
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
Surgical Approach: Intracochlear injection Fenestration: Not publicly disclosed Cochlear access: The precise cochlear access route is not publicly disclosed One or both ears; one or two administrations

Full study record View sources

OTOV101N + OTOV101C

Otovia Therapeutics

OTOF

Early clinicalActive

Who can take part: People aged 1 year and older with OTOF-related hearing loss · 1 year and older
Countries: China
Study start date: Jun 26, 2023
FDA approval: No
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
Surgical Approach: Intracochlear administration Fenestration: No separate fenestration reported; round-window membrane injection Cochlear access: Round-window membrane injection; preclinical program work used a transmastoid facial-recess corridor Cohort-dependent

Full study record View sources

SENS-501 (Audiogene)

Sensorion

OTOF

Phase 1/2Active, Not Recruiting

Who can take part: Children with severe-to-profound pre-lingual OTOF-related hearing loss · 6–31 months
Countries: Australia and France
Study start date: Jun 21, 2024
FDA approval: No
Vector: Hybrid dual AAV8 vectors encoding human OTOF transcript variant 5, with AP1-mediated reconstitution
Promoter: CMV promoter
Surgical Approach: Unilateral intracochlear injection with a proprietary delivery device Fenestration: Yes — stapes footplate (stapedotomy) Cochlear access: Round-window membrane injection; translational work describes stapedotomy pressure relief Unilateral

Full study record View sources

SKY-GJB2 (SONIX)

Skylark Bio Inc.

GJB2

Phase 1/2Actively Recruiting

Who can take part: Children with biallelic GJB2-mediated hearing loss · 9 months–7 years
Countries: United States
Study start date: May 28, 2026
FDA approval: No
Vector: AAV gene-replacement vector delivering functional GJB2 via a proprietary Skylark capsid; the capsid identity is not publicly disclosed
Promoter: Not publicly disclosed
Surgical Approach: Single unilateral intracochlear infusion via transcanal endoscopic tympanotomy using SKY-CAT Fenestration: No additional fenestration reported Cochlear access: Round-window access via transcanal endoscopic tympanotomy Unilateral

Full study record View sources