Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss

NCTID	NCT05821959 (View at clinicaltrials.gov)
Description	Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device to safely achieve the intended product performance. (Show More)
Development Status	Active
Indication	Sensorineural Hearing Loss, Bilateral
Disease Ontology Term	DOID:0110535
Compound Name	AK-OTOF
Compound Description	AAVAnc80-hOTOF
Sponsor	Akouos, Inc.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	18 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	OTOF
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracochlear
Drug Product Type	Viral vector
Target Tissue/Cell	Hair cell
Delivery System	Viral transduction
Vector Type	dual Anc80L65
Editor Type	none
Dose 1	4.1E11 vg/cochlea
Dose 2	8.1E11 vg/cochlea
Dose 3
Dose 4
Dose 5

No.of Trial Sites	8
Locations	Canada,United States,Taiwan,United Kingdom

Has US IND	True
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation
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