Gene therapy restores hearing in both ears

Gene therapy

Five children who were born with congenital hearing loss can hear in both ears after taking part in a gene therapy trial at Fudan University in Shanghai, China.

Within weeks of receiving the therapy, the children could locate the sources of sounds, and recognised speech in noisy environments. Two of the children were recorded dancing to music, the researchers reported in Nature Medicine. Read the full paper.

It is the second phase of a project that was started in January 2024, where patients were treated in one ear.

The gene therapy RRG-003 targets the otoferlin (OTOF) gene mutation causing congenital hearing loss, a condition affecting 26 million people globally. OTOF gene mutations, associated with DFNB9, a type of autosomal recessive congenital hearing loss, result in severe or profound hearing loss and speech disorders.

As the OTOF gene exceeds the loading capacity of a single adeno-associated virus (AAV), to deliver the therapy, the research team utilised a dual-vector delivery system, with two AAV vectors carrying the OTOF gene.

Ongoing gene therapy trial

In May 2024, Regeneron’s investigational gene therapy DB-OTO improved hearing to normal levels in a child (dosed at 11 months of age) with the OTOF gene mutation within 24 weeks in the ongoing Phase I/II CHORD trial.

“The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime,” said Lawrence Lustig, Chair of Columbia University’s Department of Otolaryngology – Head & Neck Surgery and a clinical trial investigator.

“With the DB-OTO CHORD trial now enrolling participants in sites across the US and Europe, we’re part of the beginning of a new era of gene therapy research that looks to create treatment options that address the root cause of profound genetic deafness.”

Diana Spencer, Senior Digital Content Editor, DDW

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free