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 unilateral administration of one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos delivery device to safely achieve the intended product performance. (Show More)
Indication	Congenital Hearing Loss
Compound Name	AAVAnc80-hOTOF
Sponsor	Akouos, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	14

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	Anc80L65
Editor Type
Dose 1	4.1 x 10^11 vg/cochlea
Dose 2	8.1 x 10^11 vg/cochlea
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,Taiwan,United Kingdom

Has US IND	True
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