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AK-OTOF
- Development stage
- Unknown
- Lead developer
- Akouos
- Modality
- Gene Therapies
- Administration
- Otic
01
Overview
**AK-OTOF** is an investigational, one-time dual-vector adeno-associated virus gene therapy developed by Akouos and Eli Lilly for autosomal-recessive otoferlin-mediated sensorineural hearing loss. The product uses two AAVAnc80 vector components that together deliver the approximately 6 kb human **OTOF** coding sequence to cochlear inner hair cells. Expression of functional otoferlin is intended to restore synaptic-vesicle release and auditory neurotransmission that are impaired by biallelic OTOF mutations. AK-OTOF is administered by local intracochlear injection using an investigational delivery device and is being evaluated in the Phase 1/2 AK-OTOF-101 trial. ([akouos.com](https://www.akouos.com/wp-content/uploads/2023/02/Preclinical-Development-of-a-Genetic-Medicine-for-Otoferlin-Gene-mediated-Hearing-Loss-AK-OTOF.pdf))
- Other names
- AAVAnc80-hOTOFAAVAnc-80-hOTOFAAVAnc 80-hOTOF
02
Targets
OTOF (Otoferlin)
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