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The study drug and study device

What is the study drug?

The study drug (AAVAnc80-hOTOF, or AK-OTOF) is an investigational medicinal product. It is an adeno-associated viral (AAV) vector gene therapy designed to deliver a working copy of the otoferlin gene to cells in the ear. With a working copy of the gene in the cells of the cochlea, the organ of hearing, the body may be able to make functional otoferlin protein and hearing may improve. The study drug will be administered during a surgical procedure using the study device.

Watch a short video provided by the American Society of Gene & Cell Therapy to learn more about gene therapy for genetic hearing loss.

Will I/my child receive the study drug?

Trial participants who meet all eligibility criteria will receive one dose of the study drug. There is no placebo in this trial.

What is the study device?

The study device is used to help deliver the study drug to the ear.

Watch a short video to learn more about the administration procedure.

Are the study drug and study device approved?

The study drug and study device are not approved by regulatory authorities like the US Food and Drug Administration (FDA), UK Medicines and Healthcare products Regulatory Agency (MHRA), or European Medicines Agency (EMA), and they can only be used in clinical research trials like the AK-OTOF-101 trial.

See if you or your child may qualify