Skip to main content
AAO-HNSF 2025 Annual Meeting & OTO EXPO Main banner
Icon Legend
This session is not in your schedule.
This session is in your schedule. Click again to remove it.

Scientific Oral Presentations

Pediatric Otolaryngology

Session: Scientific Oral Presentation 2 - Pediatric Otolaryngology

Preliminary Efficacy and Safety of AK-OTOF Gene Therapy for OTOF-mediated Hearing Loss

Sunday, October 12, 2025
8:30 AM - 8:36 AM EDT
Location: 233-234
Disclosure(s):
Chen-Chi Wu, MD, PhD:

faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

Marlan R. Hansen, MD:

faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

Introduction: The otoferlin gene (OTOF) encodes otoferlin, a protein critical for signaling at the inner hair cell synapse; individuals with mutations in OTOF typically present with congenital, severe to profound sensorineural hearing loss, with preserved otoacoustic emissions early in life. Recent advances in gene therapy and intracochlear delivery support the potential to restore hearing in individuals with OTOF-mediated hearing loss using a one-time, local administration of AK-OTOF (AAVAnc80-hOTOF). A multicenter Phase 1/2 clinical trial (NCT05821959) has been initiated to evaluate the investigational medicinal product, AK-OTOF, as well as the investigational medical device, the Akouos Delivery Device, used to administer AK-OTOF to the intracochlear space.

Methods: Participants in the AK-OTOF-101 Clinical Trial have profound hearing loss at baseline as assessed by auditory brainstem response (ABR) and receive, using a minimally invasive transcanal approach, a single intracochlear administration of AK-OTOF in one ear. Safety assessments and hearing restoration, including by ABR and behavioral audiometry testing, are assessed over the one-year trial and an additional four-year long term follow-up period.

Results: The first participant, an 11-year-old with profound congenital hearing loss, experienced restored hearing within 30 days of AK-OTOF administration, achieving behavioral thresholds of 65 to 20 dB HL. The second participant, an 8-year-old, also experienced restored hearing within 30 days of AK-OTOF administration. The surgical administration procedure and the product candidate were well tolerated, and no serious adverse events occurred as of the date of this report. Updated safety and efficacy data from these and additional participants will be presented including at least 3 months of follow-up data

Conclusions: Interim data suggest that AK-OTOF may be safely administered to patients with onset of restoration of hearing as early as one month following administration.

    Presenting Author(s)

  • CW

    Chen-Chi Wu, MD, PhD

    Professor
    National Taiwan University Hospital
    Taipei, Taipei, Taiwan (Republic of China)

    Disclosure(s):

    faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

    • Senior Author(s)

  • CW

    Chen-Chi Wu, MD, PhD

    Professor
    National Taiwan University Hospital
    Taipei, Taipei, Taiwan (Republic of China)

    Disclosure(s):

    faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

    • Co-Author(s)

  • Marlan R. Hansen, MD

    Professor and Chair
    Univ of Iowa
    Iowa City, IA, United States

    Disclosure(s):

    faculty for this accredited education activity has no relationship(s) with ineligible companies to disclose.

  • John A. Germiller, MD, PhD

    Associate Professor, Division of Pediatric Otolaryngology - Head and Neck Surgery
    Children's Hospital of Philadelphia
    Philadelphia, PA, United States

    Disclosure information not submitted.

  • Os

    On behalf of the AK-OTOF trial sponsored by Akouos and Lilly

    Sponsor
    Eli Lilly and Company
    Boston, MA, United States

    Disclosure information not submitted.