Experts from around the world have developed the first international expert consensus on gene therapy for hereditary hearing loss, stating it will be “highly valuable at the beginning of a new era in hearing loss treatment”.
They said it marked a significant step toward standardised and globally applicable guidelines for gene therapy of hearing loss.
Although recent gene therapy trials for congenital deafness had been successful, the field lacked standardised guidelines, they added.
The panel of 46 multidisciplinary experts across otology, genetics, audiology, ENT surgery, gene therapy, and hearing rehabilitation were from China, Austria, Germany, the UK, the US, Spain, Korea and Taiwan.
They developed 30 statements addressing ethical review, patient selection, diagnosis and preoperative evaluation, drug delivery, follow-up, and rehabilitation.
The consensus, published in MED A Cell Press journal on 23 October 2025, provided a critical framework for conducting safe and high-quality clinical trials, accelerating the translation from basic research to clinical practice, they added.
“This consensus provides the first globally endorsed framework for gene therapy in hereditary hearing loss,” they wrote. “It standardises clinical trial design and patient management, accelerating translation from research to practice while ensuring safety.
“The guidelines are immediately applicable to OTOF-related hearing loss and adaptable to other genetic forms.”
A modified Delphi process was conducted from March 2024 to March 2025, and after a systematic literature review, as well as integration of research and clinical expertise and experience, three iterative voting rounds (two anonymous surveys and one online consensus meeting) were performed.
Helpful for other diseases
Statements required ≥75% agreement for inclusion, with 30 evidence and expertise-based statements across six key domains achieving consensus.
“Our work developed an international expert consensus on gene therapy for hereditary HL (hearing loss), which is highly valuable at the beginning of a new era in HL treatment, providing guidance for relevant professionals and promoting the standardisation and homogeneity of gene therapy for hereditary HL across diverse regions and institutions,” the experts said.
“Importantly, our work also provides a reference framework for the design and implementation of gene therapy for other hereditary diseases.
“Future studies are needed to provide more evidence, guiding clinical decision-making and clinical practice. With the conduction of more trials in the future, this consensus will be continuously updated.”
Safety and efficacy
Senior author Dr Zheng-Yi Chen of Harvard Medical School in Cambridge, US said: “Cochlear gene therapy holds transformative potential for treating genetic hearing loss. This consensus provides essential guidance to help ensure that future clinical trials are conducted safely and effectively, at a time when the field is advancing rapidly.”
Dr Yilai Shu of Fudan University in Shanghai, China, another senior author, added: “Although no gene therapies for hearing loss have yet been commercialised, several clinical trials are already underway around the world.
“If there is no standardised consensus, differences in trial design and outcomes measurement could hinder data comparability and integration. This consensus provides a unified framework to guide current and future clinical trials and to ensure the safety, consistency, and reliability as the field advances.”
“This consensus marks a major milestone for the field of cochlear gene therapy,” says Dr Lawrence Lustig of Columbia University in New York, US, said the consensus marked a major milestone for the field of cochlear gene therapy.
“As gene therapy for hereditary hearing loss moves from concept to clinical reality, this global effort ensures that progress is driven by safety, scientific rigour, and collaboration,” he said.
The team highlighted the need for patient-centered care and respect for the diversity of perspectives within the hearing community to ensure that the field advances with both integrity and compassion for those impacted by hearing loss.
“We respect the diversity of perspectives within the deaf community,” Dr Shu said. “The final choice of whether to consider one of these treatments belongs to the adequately informed family, and their decisions should be respected.”
Several of the experts declared interests including receiving funding, working for, consulting for or being on the board of companies that are developing gene therapy for hearing loss.
