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AK-OTOF by Eli Lilly and Akouos

Also known as: AK-OTOF, AAV.OTOF, AK-101, AK-OTOF-101

Eli Lilly and Company

Primary source
Company
Eli Lilly and Company
Program Type
Therapeutic
Modality
Gene Therapy
Stage
Phase 2
Status
Active
Target Gene
OTOF
Route / Mode
Intracochlear injection
Confidence
95%
Status Confidence
Status confirmed
Evidence Strength
Strong evidence
Last Reviewed
2026-05-31
Geography
United States
Population
pediatric, adult, congenital, genetic, otof-dfnb9, pre-lingual, bilateral, profound
Trial IDs
1
Mechanism

AAV-mediated OTOF gene replacement is delivered to the cochlea to restore otoferlin function in genetic hearing loss.

Indications
Hearing LossSensorineural Hearing LossGenetic Hearing LossOTOF-Related Hearing LossAuditory Neuropathy
Notes

Lead Akouos program acquired by Lilly in 2022; Lilly publicly classified OTOF gene therapy as Phase 2 in later annual reports while the ongoing named study remains AK-OTOF-101.

Evidence & Sources
3 structured sources
clinicaltrials
Supports: canonicalName, aliases, trialIds, route, populationTags, geography
IDs: NCT05821959
Open
company
Supports: company, allCompanies, stage, status, indications
IDs: OTOF Gene Therapy
Open
company
Supports: company, allCompanies, mechanism, targetGene
IDs: AK-OTOF
Open
Key Events
2022-10-18
Lilly announced acquisition of Akouos and highlighted AK-OTOF as the lead program
2024-02-21
Lilly annual report stated Phase 2 trial initiation for OTOF gene therapy
Clinical Trials (1)
NCT05821959
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