LM220 - Congenital Hearing Loss: The Beginning of Gene Therapy Clinical Trials in the Field of Audiology

Thursday, April 18, 2024

4:45 PM – 5:45 PM EST

Location: A402-403

Earn 0.1 CEUs

Instructional Level: Intermediate

Lead Presenter(s)

Jonathon Whitton, AuD, PhD

Biotechnology Executive
Regeneron Pharmaceuticals

Disclosure(s): No financial or nonfinancial relationships to disclose.

Presenter(s)

JR

Jason Riggs, AuD, PhD

Associate Director, Clinical Sciences
Regeneron Pharmaceuticals

Disclosure(s): No financial or nonfinancial relationships to disclose.

Abstract:
Genetic hearing loss accounts for more than half of congenital deafness. Audiologists are poised to play a key role in diagnosing and managing genetic deafness. Here we will review genetic causes of hearing loss and the barriers that often prevent families from receiving a genetic diagnosis for their child. We will review the current landscape for novel precision gene therapy development to treat congenital hearing loss. Finally, we will share the latest results from Regeneron’s ongoing first-in-human gene therapy clinical trial for pediatric patients with otoferlin-related hearing loss.

Learning Objectives:

Understand genetics of hearing loss & development of potential therapeutics for hearing loss.
Understand barriers to genetic testing.
Learn about early clinical trial results from a first-in-human gene therapy clinical trial (DB-OTO).