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 a study device.

Watch the short video below to learn more about gene therapy.

Video provided by the American Society of Gene & Cell Therapy.

Will I receive the study drug?

Yes. The study is an open label, dose escalation study, with no placebo; each participant meeting all eligibility criteria will receive the study drug.

What is the study device?

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

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) or European Medicines Agency (EMA), and they can only be used in clinical research studies like the AK-OTOF-101 study.