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AAVAnc80-hOTOF via Akouos Delivery Device

Phase 1

Sensorineural Hearing Loss, Bilateral | Gene therapy | Ophthalmology |Eli Lilly and Company|Last Updated: Mar 4, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment22

FDA Designations

No designations recorded

Clinical trial landscape

AAVAnc80-hOTOF via Akouos Delivery Device · 1 trial · 1 indication

Phase 1 1
NCT05821959Gene Therapy Trial for Otoferlin Gene-mediated Hearing LossSensorineural Hearing Loss, Bilateral
RECRUITING22 Analytics
PHASE1RECRUITING
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Sensorineural Hearing Loss, BilateralUnlock trial analytics

Study Endpoints

Primary Endpoints

Frequency of Adverse Events (AEs)
through trial completion, approximately 1 year

AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery system)

Secondary Endpoints

Auditory Brainstem Response (ABR) Threshold
through trial completion, approximately 1 year
Performance of the Akouos Delivery Device and Precision Delivery Mechanism
through trial completion, approximately 1 year
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1a and Cohort 1bEXPERIMENTALCohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device Cohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
Cohort 2EXPERIMENTALCohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age
Bilateral ExpansionEXPERIMENTALBilateral Expansion: up to eight eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF).

Interventions

NameTypeDescription
AAVAnc80-hOTOF via Akouos Delivery DeviceCOMBINATION_PRODUCTAAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.
AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery MechanismCOMBINATION_PRODUCTAAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device together with the Precision Delivery Mechanism.
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Eligibility Criteria

SexALL
Healthy VolunteersNo
Study Sites9

Criteria for Inclusion: 1. Participants may be of any age, based on Cohort Criteria 2. At least two mutations in the otoferlin gene 3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR 4. Preserved distortion product otoacoustic emissions (DPOAEs) 5. Able and...

Countries:United StatesCanadaTaiwanUnited Kingdom
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Frequently asked questions about AAVAnc80-hOTOF via Akouos Delivery Device

What is AAVAnc80-hOTOF via Akouos Delivery Device used for?

AAVAnc80-hOTOF via Akouos Delivery Device is an investigational gene therapy being developed for the treatment of bilateral sensorineural hearing loss. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.

Who makes AAVAnc80-hOTOF via Akouos Delivery Device?

AAVAnc80-hOTOF via Akouos Delivery Device is being developed by Eli Lilly and Company, a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol LLY.

What phase is AAVAnc80-hOTOF via Akouos Delivery Device in?

AAVAnc80-hOTOF via Akouos Delivery Device is in Phase 1 clinical development. It is an investigational gene therapy and has not yet been approved by the FDA or other regulatory agencies.

What clinical trials is AAVAnc80-hOTOF via Akouos Delivery Device in?

AAVAnc80-hOTOF via Akouos Delivery Device is being studied in a Phase 1 clinical trial with the identifier NCT05821959. This trial is currently recruiting participants and is being conducted in the United States, Canada, Taiwan, and the United Kingdom.

Is AAVAnc80-hOTOF via Akouos Delivery Device a gene therapy?

Yes, AAVAnc80-hOTOF via Akouos Delivery Device is a gene therapy. It is being investigated for the treatment of bilateral sensorineural hearing loss, and the ongoing clinical trial is specifically focused on otoferlin gene-mediated hearing loss.